Intra-Erythrocyte Infusion of Dexamethasone Reduces Neurological Symptoms in Ataxia Teleangiectasia Patients: Results of a Phase 2 Trial View Full Text


Ontology type: schema:ScholarlyArticle      Open Access: True


Article Info

DATE

2014-12

AUTHORS

Luciana Chessa, Vincenzo Leuzzi, Alessandro Plebani, Annarosa Soresina, Roberto Micheli, Daniela D’Agnano, Tullia Venturi, Anna Molinaro, Elisa Fazzi, Mirella Marini, Pierino Ferremi Leali, Isabella Quinti, Filomena Monica Cavaliere, Gabriella Girelli, Maria Cristina Pietrogrande, Andrea Finocchi, Stefano Tabolli, Damiano Abeni, Mauro Magnani

ABSTRACT

BACKGROUND: Ataxia Teleangiectasia [AT] is a rare neurodegenerative disease characterized by early onset ataxia, oculocutaneous teleangiectasias, immunodeficiency, recurrent infections, radiosensitivity and proneness to cancer. No therapies are available for this devastating disease. Recent observational studies in few patients showed beneficial effects of short term treatment with betamethasone. To avoid the characteristic side effects of long-term administration of steroids we developed a method for encapsulation of dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EryDex) allowing slow release of dexamethasone for up to one month after dosing. Aims of the study were: the assessment of the effect of EryDex in improving neurological symptoms and adaptive behaviour of AT patients; the safety and tolerability of the therapy. METHODS: Twenty two patients (F:M=1; mean age 11.2 ± 3.5) with a confirmed diagnosis of AT and a preserved or partially supported gait were enrolled for the study. The subjects underwent for six months a monthly infusion of EryDex. Ataxia was assessed by the International Cooperative Ataxia Rating Scale (ICARS) and the adaptive behavior by Vineland Adaptive Behavior Scales (VABS). Clinical evaluations were performed at baseline and 1, 3, and 6 months. RESULTS: An improvement in ICARS (reduction of the score) was detected in the intention-to-treat (ITT) population (n=22; p=0.02) as well as in patients completing the study (per protocol PP) (n=18; p=0.01), with a mean reduction of 4 points (ITT) or 5.2 points (PP). When compared to baseline, a significant improvement were also found in VABS (increase of the score) (p<0.0001, ITT, RMANOVA), with statistically significant increases at 3 and 6 months (p<0.0001). A large inter-patient variability in the incorporation of DSP into erythrocytes was observed, with an evident positive effect of higher infusion dose on ICARS score decline. Moreover a more marked improvement was found in less neurologically impaired patients. Finally, a 19 month-extension study involving a subgroup of patients suggested that Erydex treatment can possibly delay the natural progression of the disease.EryDex was well tolerated; the most frequent side effects were common AT pathologies. CONCLUSIONS: EryDex treatment led to a significant improvement in neurological symptoms, without association with the typical steroid side effects. TRIAL REGISTRATION: Current Controlled Trial 2010-022315-19SpA. More... »

PAGES

5

References to SciGraph publications

Identifiers

URI

http://scigraph.springernature.com/pub.10.1186/1750-1172-9-5

DOI

http://dx.doi.org/10.1186/1750-1172-9-5

DIMENSIONS

https://app.dimensions.ai/details/publication/pub.1036998157

PUBMED

https://www.ncbi.nlm.nih.gov/pubmed/24405665


Indexing Status Check whether this publication has been indexed by Scopus and Web Of Science using the SN Indexing Status Tool
Incoming Citations Browse incoming citations for this publication using opencitations.net

JSON-LD is the canonical representation for SciGraph data.

TIP: You can open this SciGraph record using an external JSON-LD service: JSON-LD Playground Google SDTT

[
  {
    "@context": "https://springernature.github.io/scigraph/jsonld/sgcontext.json", 
    "about": [
      {
        "id": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/1103", 
        "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
        "name": "Clinical Sciences", 
        "type": "DefinedTerm"
      }, 
      {
        "id": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/11", 
        "inDefinedTermSet": "http://purl.org/au-research/vocabulary/anzsrc-for/2008/", 
        "name": "Medical and Health Sciences", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Adolescent", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Ataxia Telangiectasia", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Child", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Dexamethasone", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Disease Progression", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Erythrocytes", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Female", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Humans", 
        "type": "DefinedTerm"
      }, 
      {
        "inDefinedTermSet": "https://www.nlm.nih.gov/mesh/", 
        "name": "Male", 
        "type": "DefinedTerm"
      }
    ], 
    "author": [
      {
        "affiliation": {
          "alternateName": "Sapienza University of Rome", 
          "id": "https://www.grid.ac/institutes/grid.7841.a", 
          "name": [
            "Department of Clinical and Molecular Medicine, Sapienza Universit\u00e0 di Roma, Via di Grottarossa 1035, 00189, Roma, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Chessa", 
        "givenName": "Luciana", 
        "id": "sg:person.01210613030.05", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01210613030.05"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "Sapienza University of Rome", 
          "id": "https://www.grid.ac/institutes/grid.7841.a", 
          "name": [
            "Department of Pediatrics and Child Neurology and Psychiatry, Sapienza Universit\u00e0 di Roma, via dei Sabelli 108, 00185, Roma, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Leuzzi", 
        "givenName": "Vincenzo", 
        "id": "sg:person.0612121222.23", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0612121222.23"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "name": [
            "Department of Clinical and Experimental Sciences, Pediatrics Clinic and Institute of Molecular Medicine A. Nocivelli, Spedali Civili and University of Brescia, Piazza Spedali Civili, 1 25123, Brescia, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Plebani", 
        "givenName": "Alessandro", 
        "id": "sg:person.01111477410.08", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01111477410.08"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "name": [
            "Department of Clinical and Experimental Sciences, Pediatrics Clinic and Institute of Molecular Medicine A. Nocivelli, Spedali Civili and University of Brescia, Piazza Spedali Civili, 1 25123, Brescia, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Soresina", 
        "givenName": "Annarosa", 
        "id": "sg:person.01021536037.33", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01021536037.33"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "name": [
            "Unit of Child Neurology and Psychiatry, Spedali Civili and Universit\u00e0 di Brescia, Piazza Spedali Civili, 1 25123, Brescia, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Micheli", 
        "givenName": "Roberto", 
        "id": "sg:person.01053453435.12", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01053453435.12"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "Sapienza University of Rome", 
          "id": "https://www.grid.ac/institutes/grid.7841.a", 
          "name": [
            "Department of Pediatrics and Child Neurology and Psychiatry, Sapienza Universit\u00e0 di Roma, via dei Sabelli 108, 00185, Roma, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "D\u2019Agnano", 
        "givenName": "Daniela", 
        "id": "sg:person.0774463022.00", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0774463022.00"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "Sapienza University of Rome", 
          "id": "https://www.grid.ac/institutes/grid.7841.a", 
          "name": [
            "Department of Pediatrics and Child Neurology and Psychiatry, Sapienza Universit\u00e0 di Roma, via dei Sabelli 108, 00185, Roma, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Venturi", 
        "givenName": "Tullia", 
        "id": "sg:person.01300034772.90", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01300034772.90"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "University of Brescia", 
          "id": "https://www.grid.ac/institutes/grid.7637.5", 
          "name": [
            "School in Reproductive and Developmental Science, Universit\u00e0 di Trieste and Universit\u00e0 di Brescia, Brescia, Piazzale Spedali Civili, 1 25123, Brescia, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Molinaro", 
        "givenName": "Anna", 
        "id": "sg:person.0776277160.98", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0776277160.98"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "name": [
            "Unit of Child Neurology and Psychiatry, Spedali Civili and Universit\u00e0 di Brescia, Piazza Spedali Civili, 1 25123, Brescia, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Fazzi", 
        "givenName": "Elisa", 
        "id": "sg:person.01235060202.38", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01235060202.38"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "name": [
            "Department of Clinical and Experimental Sciences, Pediatrics Clinic and Institute of Molecular Medicine A. Nocivelli, Spedali Civili and University of Brescia, Piazza Spedali Civili, 1 25123, Brescia, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Marini", 
        "givenName": "Mirella", 
        "id": "sg:person.01074502550.02", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01074502550.02"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "name": [
            "Department of Clinical and Experimental Sciences, Pediatrics Clinic and Institute of Molecular Medicine A. Nocivelli, Spedali Civili and University of Brescia, Piazza Spedali Civili, 1 25123, Brescia, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Ferremi Leali", 
        "givenName": "Pierino", 
        "id": "sg:person.012224446741.87", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.012224446741.87"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "Sapienza University of Rome", 
          "id": "https://www.grid.ac/institutes/grid.7841.a", 
          "name": [
            "Department of Molecular Medicine, Sapienza Universit\u00e0 di Roma, Viale dell\u2019Universit\u00e0 37, 00186, Roma, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Quinti", 
        "givenName": "Isabella", 
        "id": "sg:person.01235174604.07", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01235174604.07"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "Sapienza University of Rome", 
          "id": "https://www.grid.ac/institutes/grid.7841.a", 
          "name": [
            "Department of Molecular Medicine, Sapienza Universit\u00e0 di Roma, Viale dell\u2019Universit\u00e0 37, 00186, Roma, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Cavaliere", 
        "givenName": "Filomena Monica", 
        "id": "sg:person.01130036136.23", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01130036136.23"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "Sapienza University of Rome", 
          "id": "https://www.grid.ac/institutes/grid.7841.a", 
          "name": [
            "Department of Molecular Medicine, Sapienza Universit\u00e0 di Roma, Viale dell\u2019Universit\u00e0 37, 00186, Roma, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Girelli", 
        "givenName": "Gabriella", 
        "id": "sg:person.01361735776.32", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01361735776.32"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "University of Milan", 
          "id": "https://www.grid.ac/institutes/grid.4708.b", 
          "name": [
            "Department of Pediatrics, Universit\u00e0 di Milano, Fondazione IRCCS Ca\u2019 Granda, via Commenda 9, 20122, Milano, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Pietrogrande", 
        "givenName": "Maria Cristina", 
        "id": "sg:person.0712513372.02", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0712513372.02"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "name": [
            "Department of Pediatrics, Ospedale Pediatrico Bambino Ges\u00f9 and Universit\u00e0 di Tor Vergata, Piazza di San Onofrio 4, 00165, Roma, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Finocchi", 
        "givenName": "Andrea", 
        "id": "sg:person.0733032004.10", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0733032004.10"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "Istituto Dermopatico dell'Immacolata", 
          "id": "https://www.grid.ac/institutes/grid.419457.a", 
          "name": [
            "Istituto Dermopatico Immacolata, Via Monti Creta, 104, 00167, Roma, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Tabolli", 
        "givenName": "Stefano", 
        "id": "sg:person.0750701253.03", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0750701253.03"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "Istituto Dermopatico dell'Immacolata", 
          "id": "https://www.grid.ac/institutes/grid.419457.a", 
          "name": [
            "Istituto Dermopatico Immacolata, Via Monti Creta, 104, 00167, Roma, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Abeni", 
        "givenName": "Damiano", 
        "id": "sg:person.0666726716.85", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0666726716.85"
        ], 
        "type": "Person"
      }, 
      {
        "affiliation": {
          "alternateName": "University of Urbino", 
          "id": "https://www.grid.ac/institutes/grid.12711.34", 
          "name": [
            "Department of Biomolecular Sciences, Universit\u00e0 di Urbino \u201cCarlo Bo\u201d, Via Saffi 2, Erydel S.p.A, Via Sasso, 61029, 61029, Urbino, Urbino, Italy, Italy"
          ], 
          "type": "Organization"
        }, 
        "familyName": "Magnani", 
        "givenName": "Mauro", 
        "id": "sg:person.0752252542.98", 
        "sameAs": [
          "https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0752252542.98"
        ], 
        "type": "Person"
      }
    ], 
    "citation": [
      {
        "id": "https://doi.org/10.1016/j.bcmd.2004.04.004", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1002331703"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1111/j.1468-1331.2010.03203.x", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1002417286"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1111/j.1470-8744.1998.tb00505.x", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1004789617"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1111/j.1468-1331.2009.02600.x", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1007007344"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1111/j.1468-1331.2009.02600.x", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1007007344"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1002/mds.25126", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1009326820"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1074/jbc.m112.344473", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1012734720"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1002/mds.21543", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1014132319"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/0005-2736(90)90272-p", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1018056533"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/0005-2736(90)90272-p", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1018056533"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1136/adc.2006.094268", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1027065426"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1111/j.1572-0241.2005.41412.x", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1027466906", 
          "https://doi.org/10.1111/j.1572-0241.2005.41412.x"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1111/j.1572-0241.2005.41412.x", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1027466906", 
          "https://doi.org/10.1111/j.1572-0241.2005.41412.x"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1002/mpr.282", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1028514340"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1002/mpr.282", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1028514340"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1097/mpg.0b013e3180320667", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1028955820"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1097/mpg.0b013e3180320667", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1028955820"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1186/1471-2431-6-17", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1030404156", 
          "https://doi.org/10.1186/1471-2431-6-17"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1007/978-3-211-69500-5_49", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1030473349", 
          "https://doi.org/10.1007/978-3-211-69500-5_49"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1111/j.1468-1331.2008.02060.x", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1033349556"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1111/j.1572-0241.2008.02103.x", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1035274192", 
          "https://doi.org/10.1111/j.1572-0241.2008.02103.x"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1177/0883073812441999", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1037362094"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1177/0883073812441999", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1037362094"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/35043058", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1037972283", 
          "https://doi.org/10.1038/35043058"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/35043058", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1037972283", 
          "https://doi.org/10.1038/35043058"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "sg:pub.10.1038/35043058", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1037972283", 
          "https://doi.org/10.1038/35043058"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1002/mds.20781", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1039904839"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1002/mds.20781", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1039904839"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1001/archneur.63.10.1479", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1044242012"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/s0022-510x(96)00231-6", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1044787153"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1002/iub.478", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1047203985"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1001/archneur.63.10.1469", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1047368769"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1016/0005-2760(96)00067-7", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1052965762"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1212/wnl.0b013e318225ab2e", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1064356304"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1212/wnl.0b013e318225ab2e", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1064356304"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1212/wnl.0b013e318225ab2e", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1064356304"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1212/wnl.0b013e3182494d51", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1064356788"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1212/wnl.0b013e3182494d51", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1064356788"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1212/wnl.0b013e3182494d51", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1064356788"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://doi.org/10.1212/wnl.54.7.1505", 
        "sameAs": [
          "https://app.dimensions.ai/details/publication/pub.1064378560"
        ], 
        "type": "CreativeWork"
      }, 
      {
        "id": "https://app.dimensions.ai/details/publication/pub.1078688731", 
        "type": "CreativeWork"
      }
    ], 
    "datePublished": "2014-12", 
    "datePublishedReg": "2014-12-01", 
    "description": "BACKGROUND: Ataxia Teleangiectasia [AT] is a rare neurodegenerative disease characterized by early onset ataxia, oculocutaneous teleangiectasias, immunodeficiency, recurrent infections, radiosensitivity and proneness to cancer. No therapies are available for this devastating disease. Recent observational studies in few patients showed beneficial effects of short term treatment with betamethasone. To avoid the characteristic side effects of long-term administration of steroids we developed a method for encapsulation of dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EryDex) allowing slow release of dexamethasone for up to one month after dosing. Aims of the study were: the assessment of the effect of EryDex in improving neurological symptoms and adaptive behaviour of AT patients; the safety and tolerability of the therapy.\nMETHODS: Twenty two patients (F:M=1; mean age 11.2 \u00b1 3.5) with a confirmed diagnosis of AT and a preserved or partially supported gait were enrolled for the study. The subjects underwent for six months a monthly infusion of EryDex. Ataxia was assessed by the International Cooperative Ataxia Rating Scale (ICARS) and the adaptive behavior by Vineland Adaptive Behavior Scales (VABS). Clinical evaluations were performed at baseline and 1, 3, and 6 months.\nRESULTS: An improvement in ICARS (reduction of the score) was detected in the intention-to-treat (ITT) population (n=22; p=0.02) as well as in patients completing the study (per protocol PP) (n=18; p=0.01), with a mean reduction of 4 points (ITT) or 5.2 points (PP). When compared to baseline, a significant improvement were also found in VABS (increase of the score) (p<0.0001, ITT, RMANOVA), with statistically significant increases at 3 and 6 months (p<0.0001). A large inter-patient variability in the incorporation of DSP into erythrocytes was observed, with an evident positive effect of higher infusion dose on ICARS score decline. Moreover a more marked improvement was found in less neurologically impaired patients. Finally, a 19 month-extension study involving a subgroup of patients suggested that Erydex treatment can possibly delay the natural progression of the disease.EryDex was well tolerated; the most frequent side effects were common AT pathologies.\nCONCLUSIONS: EryDex treatment led to a significant improvement in neurological symptoms, without association with the typical steroid side effects.\nTRIAL REGISTRATION: Current Controlled Trial 2010-022315-19SpA.", 
    "genre": "research_article", 
    "id": "sg:pub.10.1186/1750-1172-9-5", 
    "inLanguage": [
      "en"
    ], 
    "isAccessibleForFree": true, 
    "isPartOf": [
      {
        "id": "sg:journal.1036535", 
        "issn": [
          "1750-1172"
        ], 
        "name": "Orphanet Journal of Rare Diseases", 
        "type": "Periodical"
      }, 
      {
        "issueNumber": "1", 
        "type": "PublicationIssue"
      }, 
      {
        "type": "PublicationVolume", 
        "volumeNumber": "9"
      }
    ], 
    "name": "Intra-Erythrocyte Infusion of Dexamethasone Reduces Neurological Symptoms in Ataxia Teleangiectasia Patients: Results of a Phase 2 Trial", 
    "pagination": "5", 
    "productId": [
      {
        "name": "readcube_id", 
        "type": "PropertyValue", 
        "value": [
          "b03f2298e28e6119e1c70eb9e8eee8ead0db825d4dfb337ed4562ca444ab38e1"
        ]
      }, 
      {
        "name": "pubmed_id", 
        "type": "PropertyValue", 
        "value": [
          "24405665"
        ]
      }, 
      {
        "name": "nlm_unique_id", 
        "type": "PropertyValue", 
        "value": [
          "101266602"
        ]
      }, 
      {
        "name": "doi", 
        "type": "PropertyValue", 
        "value": [
          "10.1186/1750-1172-9-5"
        ]
      }, 
      {
        "name": "dimensions_id", 
        "type": "PropertyValue", 
        "value": [
          "pub.1036998157"
        ]
      }
    ], 
    "sameAs": [
      "https://doi.org/10.1186/1750-1172-9-5", 
      "https://app.dimensions.ai/details/publication/pub.1036998157"
    ], 
    "sdDataset": "articles", 
    "sdDatePublished": "2019-04-10T21:37", 
    "sdLicense": "https://scigraph.springernature.com/explorer/license/", 
    "sdPublisher": {
      "name": "Springer Nature - SN SciGraph project", 
      "type": "Organization"
    }, 
    "sdSource": "s3://com-uberresearch-data-dimensions-target-20181106-alternative/cleanup/v134/2549eaecd7973599484d7c17b260dba0a4ecb94b/merge/v9/a6c9fde33151104705d4d7ff012ea9563521a3ce/jats-lookup/v90/0000000001_0000000264/records_8687_00000514.jsonl", 
    "type": "ScholarlyArticle", 
    "url": "http://link.springer.com/10.1186%2F1750-1172-9-5"
  }
]
 

Download the RDF metadata as:  json-ld nt turtle xml License info

HOW TO GET THIS DATA PROGRAMMATICALLY:

JSON-LD is a popular format for linked data which is fully compatible with JSON.

curl -H 'Accept: application/ld+json' 'https://scigraph.springernature.com/pub.10.1186/1750-1172-9-5'

N-Triples is a line-based linked data format ideal for batch operations.

curl -H 'Accept: application/n-triples' 'https://scigraph.springernature.com/pub.10.1186/1750-1172-9-5'

Turtle is a human-readable linked data format.

curl -H 'Accept: text/turtle' 'https://scigraph.springernature.com/pub.10.1186/1750-1172-9-5'

RDF/XML is a standard XML format for linked data.

curl -H 'Accept: application/rdf+xml' 'https://scigraph.springernature.com/pub.10.1186/1750-1172-9-5'


 

This table displays all metadata directly associated to this object as RDF triples.

346 TRIPLES      21 PREDICATES      66 URIs      30 LITERALS      18 BLANK NODES

Subject Predicate Object
1 sg:pub.10.1186/1750-1172-9-5 schema:about N0d5e3f8e15f4408a87cbbb5d2a3368e8
2 N13294b4c3ec24f5aaecd7c77bed35032
3 N1e7ec0dec22e4a9993a2de6385585432
4 N218a374edceb4dd1bbc0ab9353ad5a5e
5 N534205049b254102969ded5d36b807c7
6 N5ad53920a6f8455ba86071a2cdbf474f
7 N6f33993d65394667a2366b572f6a9eb6
8 Nd71f00c537af4f818dcee6f21f3e389e
9 Nfb6e49b7593c4ba9a69f672eb4933158
10 anzsrc-for:11
11 anzsrc-for:1103
12 schema:author Ncdd52cc2f9714da2ab36b62933117d3c
13 schema:citation sg:pub.10.1007/978-3-211-69500-5_49
14 sg:pub.10.1038/35043058
15 sg:pub.10.1111/j.1572-0241.2005.41412.x
16 sg:pub.10.1111/j.1572-0241.2008.02103.x
17 sg:pub.10.1186/1471-2431-6-17
18 https://app.dimensions.ai/details/publication/pub.1078688731
19 https://doi.org/10.1001/archneur.63.10.1469
20 https://doi.org/10.1001/archneur.63.10.1479
21 https://doi.org/10.1002/iub.478
22 https://doi.org/10.1002/mds.20781
23 https://doi.org/10.1002/mds.21543
24 https://doi.org/10.1002/mds.25126
25 https://doi.org/10.1002/mpr.282
26 https://doi.org/10.1016/0005-2736(90)90272-p
27 https://doi.org/10.1016/0005-2760(96)00067-7
28 https://doi.org/10.1016/j.bcmd.2004.04.004
29 https://doi.org/10.1016/s0022-510x(96)00231-6
30 https://doi.org/10.1074/jbc.m112.344473
31 https://doi.org/10.1097/mpg.0b013e3180320667
32 https://doi.org/10.1111/j.1468-1331.2008.02060.x
33 https://doi.org/10.1111/j.1468-1331.2009.02600.x
34 https://doi.org/10.1111/j.1468-1331.2010.03203.x
35 https://doi.org/10.1111/j.1470-8744.1998.tb00505.x
36 https://doi.org/10.1136/adc.2006.094268
37 https://doi.org/10.1177/0883073812441999
38 https://doi.org/10.1212/wnl.0b013e318225ab2e
39 https://doi.org/10.1212/wnl.0b013e3182494d51
40 https://doi.org/10.1212/wnl.54.7.1505
41 schema:datePublished 2014-12
42 schema:datePublishedReg 2014-12-01
43 schema:description BACKGROUND: Ataxia Teleangiectasia [AT] is a rare neurodegenerative disease characterized by early onset ataxia, oculocutaneous teleangiectasias, immunodeficiency, recurrent infections, radiosensitivity and proneness to cancer. No therapies are available for this devastating disease. Recent observational studies in few patients showed beneficial effects of short term treatment with betamethasone. To avoid the characteristic side effects of long-term administration of steroids we developed a method for encapsulation of dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EryDex) allowing slow release of dexamethasone for up to one month after dosing. Aims of the study were: the assessment of the effect of EryDex in improving neurological symptoms and adaptive behaviour of AT patients; the safety and tolerability of the therapy. METHODS: Twenty two patients (F:M=1; mean age 11.2 ± 3.5) with a confirmed diagnosis of AT and a preserved or partially supported gait were enrolled for the study. The subjects underwent for six months a monthly infusion of EryDex. Ataxia was assessed by the International Cooperative Ataxia Rating Scale (ICARS) and the adaptive behavior by Vineland Adaptive Behavior Scales (VABS). Clinical evaluations were performed at baseline and 1, 3, and 6 months. RESULTS: An improvement in ICARS (reduction of the score) was detected in the intention-to-treat (ITT) population (n=22; p=0.02) as well as in patients completing the study (per protocol PP) (n=18; p=0.01), with a mean reduction of 4 points (ITT) or 5.2 points (PP). When compared to baseline, a significant improvement were also found in VABS (increase of the score) (p<0.0001, ITT, RMANOVA), with statistically significant increases at 3 and 6 months (p<0.0001). A large inter-patient variability in the incorporation of DSP into erythrocytes was observed, with an evident positive effect of higher infusion dose on ICARS score decline. Moreover a more marked improvement was found in less neurologically impaired patients. Finally, a 19 month-extension study involving a subgroup of patients suggested that Erydex treatment can possibly delay the natural progression of the disease.EryDex was well tolerated; the most frequent side effects were common AT pathologies. CONCLUSIONS: EryDex treatment led to a significant improvement in neurological symptoms, without association with the typical steroid side effects. TRIAL REGISTRATION: Current Controlled Trial 2010-022315-19SpA.
44 schema:genre research_article
45 schema:inLanguage en
46 schema:isAccessibleForFree true
47 schema:isPartOf N2429e52fd95f480a88b8d74b5c95f2a2
48 N6d8dad3f9df34b27890438df9ee9a5f8
49 sg:journal.1036535
50 schema:name Intra-Erythrocyte Infusion of Dexamethasone Reduces Neurological Symptoms in Ataxia Teleangiectasia Patients: Results of a Phase 2 Trial
51 schema:pagination 5
52 schema:productId N908d7c4495634e4098bb149721ab9547
53 Na4787ac10cfd4398912fe4b2b9260a89
54 Na71643dbb5f94363a0ce0c04532f0a48
55 Nb91e78f67fe741278d0b1310592098bc
56 Nfcd56af3157c4ad9b9481d9675ae39e5
57 schema:sameAs https://app.dimensions.ai/details/publication/pub.1036998157
58 https://doi.org/10.1186/1750-1172-9-5
59 schema:sdDatePublished 2019-04-10T21:37
60 schema:sdLicense https://scigraph.springernature.com/explorer/license/
61 schema:sdPublisher Ne34ac7e5dd744a5fb855c06c987eca05
62 schema:url http://link.springer.com/10.1186%2F1750-1172-9-5
63 sgo:license sg:explorer/license/
64 sgo:sdDataset articles
65 rdf:type schema:ScholarlyArticle
66 N002d791a5f9b4090aaf394a9232ef9ed rdf:first sg:person.01235060202.38
67 rdf:rest N45933cd6684c46619f35aab70f6a6e83
68 N056fcb26be334e0eb406f802a11d9725 schema:name Unit of Child Neurology and Psychiatry, Spedali Civili and Università di Brescia, Piazza Spedali Civili, 1 25123, Brescia, Italy
69 rdf:type schema:Organization
70 N0a67a0fbeb6c4fe586946cdbc6ab0971 rdf:first sg:person.0733032004.10
71 rdf:rest N48dca40df4bf44179be9921d76316a9e
72 N0d5e3f8e15f4408a87cbbb5d2a3368e8 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
73 schema:name Dexamethasone
74 rdf:type schema:DefinedTerm
75 N13294b4c3ec24f5aaecd7c77bed35032 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
76 schema:name Erythrocytes
77 rdf:type schema:DefinedTerm
78 N135681a1dab140cdb0533e83df3257ba rdf:first sg:person.0774463022.00
79 rdf:rest N966f5eaa1ae141b89ec9f77fcf05bd34
80 N1e7ec0dec22e4a9993a2de6385585432 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
81 schema:name Humans
82 rdf:type schema:DefinedTerm
83 N218a374edceb4dd1bbc0ab9353ad5a5e schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
84 schema:name Ataxia Telangiectasia
85 rdf:type schema:DefinedTerm
86 N2429e52fd95f480a88b8d74b5c95f2a2 schema:issueNumber 1
87 rdf:type schema:PublicationIssue
88 N2c28f170ded841d995a4967419843a2e rdf:first sg:person.01021536037.33
89 rdf:rest Nbded4572ec9e45c4bc827422aae49402
90 N305d0131057442f2bcb7b7cb9f98c729 schema:name Department of Clinical and Experimental Sciences, Pediatrics Clinic and Institute of Molecular Medicine A. Nocivelli, Spedali Civili and University of Brescia, Piazza Spedali Civili, 1 25123, Brescia, Italy
91 rdf:type schema:Organization
92 N30ee340b39024af9b0759cf7378abecc rdf:first sg:person.01130036136.23
93 rdf:rest Ndf7fb96e347e4404b8274ac6fbddee85
94 N3664b0a4cb2e47459cabaf3a91ad3281 rdf:first sg:person.01235174604.07
95 rdf:rest N30ee340b39024af9b0759cf7378abecc
96 N36719246b8784462bc233a8cb07013ec schema:name Unit of Child Neurology and Psychiatry, Spedali Civili and Università di Brescia, Piazza Spedali Civili, 1 25123, Brescia, Italy
97 rdf:type schema:Organization
98 N45933cd6684c46619f35aab70f6a6e83 rdf:first sg:person.01074502550.02
99 rdf:rest N47eccd452bf944d7a2410005bc43f138
100 N47eccd452bf944d7a2410005bc43f138 rdf:first sg:person.012224446741.87
101 rdf:rest N3664b0a4cb2e47459cabaf3a91ad3281
102 N48dca40df4bf44179be9921d76316a9e rdf:first sg:person.0750701253.03
103 rdf:rest N9164242c86194db687023e8fafecb12c
104 N5058641295a44eed93ee8520b78234e0 schema:name Department of Clinical and Experimental Sciences, Pediatrics Clinic and Institute of Molecular Medicine A. Nocivelli, Spedali Civili and University of Brescia, Piazza Spedali Civili, 1 25123, Brescia, Italy
105 rdf:type schema:Organization
106 N52c09fa79a134667853c7c21bbfe8729 rdf:first sg:person.0612121222.23
107 rdf:rest N61ae6bf9816d4582a02ab48532824491
108 N534205049b254102969ded5d36b807c7 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
109 schema:name Female
110 rdf:type schema:DefinedTerm
111 N5ad53920a6f8455ba86071a2cdbf474f schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
112 schema:name Male
113 rdf:type schema:DefinedTerm
114 N61ae6bf9816d4582a02ab48532824491 rdf:first sg:person.01111477410.08
115 rdf:rest N2c28f170ded841d995a4967419843a2e
116 N61b42b904c754009b8c11dc8bffe1411 schema:name Department of Clinical and Experimental Sciences, Pediatrics Clinic and Institute of Molecular Medicine A. Nocivelli, Spedali Civili and University of Brescia, Piazza Spedali Civili, 1 25123, Brescia, Italy
117 rdf:type schema:Organization
118 N6d8dad3f9df34b27890438df9ee9a5f8 schema:volumeNumber 9
119 rdf:type schema:PublicationVolume
120 N6f33993d65394667a2366b572f6a9eb6 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
121 schema:name Child
122 rdf:type schema:DefinedTerm
123 N7d987049554c47d68d5d915c3863054a schema:name Department of Pediatrics, Ospedale Pediatrico Bambino Gesù and Università di Tor Vergata, Piazza di San Onofrio 4, 00165, Roma, Italy
124 rdf:type schema:Organization
125 N908d7c4495634e4098bb149721ab9547 schema:name dimensions_id
126 schema:value pub.1036998157
127 rdf:type schema:PropertyValue
128 N9164242c86194db687023e8fafecb12c rdf:first sg:person.0666726716.85
129 rdf:rest Nc2b1ceb5898043e2b9c1f7961e2ba56e
130 N966f5eaa1ae141b89ec9f77fcf05bd34 rdf:first sg:person.01300034772.90
131 rdf:rest Nf7b7089d08ac45708dd53e4f1a370104
132 N975a196269234de4bafb783eda138ad1 schema:name Department of Clinical and Experimental Sciences, Pediatrics Clinic and Institute of Molecular Medicine A. Nocivelli, Spedali Civili and University of Brescia, Piazza Spedali Civili, 1 25123, Brescia, Italy
133 rdf:type schema:Organization
134 N9a1308f588a14c45af1ea8c30f0b63a0 rdf:first sg:person.0712513372.02
135 rdf:rest N0a67a0fbeb6c4fe586946cdbc6ab0971
136 Na4787ac10cfd4398912fe4b2b9260a89 schema:name readcube_id
137 schema:value b03f2298e28e6119e1c70eb9e8eee8ead0db825d4dfb337ed4562ca444ab38e1
138 rdf:type schema:PropertyValue
139 Na71643dbb5f94363a0ce0c04532f0a48 schema:name doi
140 schema:value 10.1186/1750-1172-9-5
141 rdf:type schema:PropertyValue
142 Nb91e78f67fe741278d0b1310592098bc schema:name pubmed_id
143 schema:value 24405665
144 rdf:type schema:PropertyValue
145 Nbded4572ec9e45c4bc827422aae49402 rdf:first sg:person.01053453435.12
146 rdf:rest N135681a1dab140cdb0533e83df3257ba
147 Nc2b1ceb5898043e2b9c1f7961e2ba56e rdf:first sg:person.0752252542.98
148 rdf:rest rdf:nil
149 Ncdd52cc2f9714da2ab36b62933117d3c rdf:first sg:person.01210613030.05
150 rdf:rest N52c09fa79a134667853c7c21bbfe8729
151 Nd71f00c537af4f818dcee6f21f3e389e schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
152 schema:name Disease Progression
153 rdf:type schema:DefinedTerm
154 Ndf7fb96e347e4404b8274ac6fbddee85 rdf:first sg:person.01361735776.32
155 rdf:rest N9a1308f588a14c45af1ea8c30f0b63a0
156 Ne34ac7e5dd744a5fb855c06c987eca05 schema:name Springer Nature - SN SciGraph project
157 rdf:type schema:Organization
158 Nf7b7089d08ac45708dd53e4f1a370104 rdf:first sg:person.0776277160.98
159 rdf:rest N002d791a5f9b4090aaf394a9232ef9ed
160 Nfb6e49b7593c4ba9a69f672eb4933158 schema:inDefinedTermSet https://www.nlm.nih.gov/mesh/
161 schema:name Adolescent
162 rdf:type schema:DefinedTerm
163 Nfcd56af3157c4ad9b9481d9675ae39e5 schema:name nlm_unique_id
164 schema:value 101266602
165 rdf:type schema:PropertyValue
166 anzsrc-for:11 schema:inDefinedTermSet anzsrc-for:
167 schema:name Medical and Health Sciences
168 rdf:type schema:DefinedTerm
169 anzsrc-for:1103 schema:inDefinedTermSet anzsrc-for:
170 schema:name Clinical Sciences
171 rdf:type schema:DefinedTerm
172 sg:journal.1036535 schema:issn 1750-1172
173 schema:name Orphanet Journal of Rare Diseases
174 rdf:type schema:Periodical
175 sg:person.01021536037.33 schema:affiliation N975a196269234de4bafb783eda138ad1
176 schema:familyName Soresina
177 schema:givenName Annarosa
178 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01021536037.33
179 rdf:type schema:Person
180 sg:person.01053453435.12 schema:affiliation N056fcb26be334e0eb406f802a11d9725
181 schema:familyName Micheli
182 schema:givenName Roberto
183 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01053453435.12
184 rdf:type schema:Person
185 sg:person.01074502550.02 schema:affiliation N61b42b904c754009b8c11dc8bffe1411
186 schema:familyName Marini
187 schema:givenName Mirella
188 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01074502550.02
189 rdf:type schema:Person
190 sg:person.01111477410.08 schema:affiliation N305d0131057442f2bcb7b7cb9f98c729
191 schema:familyName Plebani
192 schema:givenName Alessandro
193 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01111477410.08
194 rdf:type schema:Person
195 sg:person.01130036136.23 schema:affiliation https://www.grid.ac/institutes/grid.7841.a
196 schema:familyName Cavaliere
197 schema:givenName Filomena Monica
198 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01130036136.23
199 rdf:type schema:Person
200 sg:person.01210613030.05 schema:affiliation https://www.grid.ac/institutes/grid.7841.a
201 schema:familyName Chessa
202 schema:givenName Luciana
203 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01210613030.05
204 rdf:type schema:Person
205 sg:person.012224446741.87 schema:affiliation N5058641295a44eed93ee8520b78234e0
206 schema:familyName Ferremi Leali
207 schema:givenName Pierino
208 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.012224446741.87
209 rdf:type schema:Person
210 sg:person.01235060202.38 schema:affiliation N36719246b8784462bc233a8cb07013ec
211 schema:familyName Fazzi
212 schema:givenName Elisa
213 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01235060202.38
214 rdf:type schema:Person
215 sg:person.01235174604.07 schema:affiliation https://www.grid.ac/institutes/grid.7841.a
216 schema:familyName Quinti
217 schema:givenName Isabella
218 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01235174604.07
219 rdf:type schema:Person
220 sg:person.01300034772.90 schema:affiliation https://www.grid.ac/institutes/grid.7841.a
221 schema:familyName Venturi
222 schema:givenName Tullia
223 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01300034772.90
224 rdf:type schema:Person
225 sg:person.01361735776.32 schema:affiliation https://www.grid.ac/institutes/grid.7841.a
226 schema:familyName Girelli
227 schema:givenName Gabriella
228 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.01361735776.32
229 rdf:type schema:Person
230 sg:person.0612121222.23 schema:affiliation https://www.grid.ac/institutes/grid.7841.a
231 schema:familyName Leuzzi
232 schema:givenName Vincenzo
233 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0612121222.23
234 rdf:type schema:Person
235 sg:person.0666726716.85 schema:affiliation https://www.grid.ac/institutes/grid.419457.a
236 schema:familyName Abeni
237 schema:givenName Damiano
238 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0666726716.85
239 rdf:type schema:Person
240 sg:person.0712513372.02 schema:affiliation https://www.grid.ac/institutes/grid.4708.b
241 schema:familyName Pietrogrande
242 schema:givenName Maria Cristina
243 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0712513372.02
244 rdf:type schema:Person
245 sg:person.0733032004.10 schema:affiliation N7d987049554c47d68d5d915c3863054a
246 schema:familyName Finocchi
247 schema:givenName Andrea
248 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0733032004.10
249 rdf:type schema:Person
250 sg:person.0750701253.03 schema:affiliation https://www.grid.ac/institutes/grid.419457.a
251 schema:familyName Tabolli
252 schema:givenName Stefano
253 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0750701253.03
254 rdf:type schema:Person
255 sg:person.0752252542.98 schema:affiliation https://www.grid.ac/institutes/grid.12711.34
256 schema:familyName Magnani
257 schema:givenName Mauro
258 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0752252542.98
259 rdf:type schema:Person
260 sg:person.0774463022.00 schema:affiliation https://www.grid.ac/institutes/grid.7841.a
261 schema:familyName D’Agnano
262 schema:givenName Daniela
263 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0774463022.00
264 rdf:type schema:Person
265 sg:person.0776277160.98 schema:affiliation https://www.grid.ac/institutes/grid.7637.5
266 schema:familyName Molinaro
267 schema:givenName Anna
268 schema:sameAs https://app.dimensions.ai/discover/publication?and_facet_researcher=ur.0776277160.98
269 rdf:type schema:Person
270 sg:pub.10.1007/978-3-211-69500-5_49 schema:sameAs https://app.dimensions.ai/details/publication/pub.1030473349
271 https://doi.org/10.1007/978-3-211-69500-5_49
272 rdf:type schema:CreativeWork
273 sg:pub.10.1038/35043058 schema:sameAs https://app.dimensions.ai/details/publication/pub.1037972283
274 https://doi.org/10.1038/35043058
275 rdf:type schema:CreativeWork
276 sg:pub.10.1111/j.1572-0241.2005.41412.x schema:sameAs https://app.dimensions.ai/details/publication/pub.1027466906
277 https://doi.org/10.1111/j.1572-0241.2005.41412.x
278 rdf:type schema:CreativeWork
279 sg:pub.10.1111/j.1572-0241.2008.02103.x schema:sameAs https://app.dimensions.ai/details/publication/pub.1035274192
280 https://doi.org/10.1111/j.1572-0241.2008.02103.x
281 rdf:type schema:CreativeWork
282 sg:pub.10.1186/1471-2431-6-17 schema:sameAs https://app.dimensions.ai/details/publication/pub.1030404156
283 https://doi.org/10.1186/1471-2431-6-17
284 rdf:type schema:CreativeWork
285 https://app.dimensions.ai/details/publication/pub.1078688731 schema:CreativeWork
286 https://doi.org/10.1001/archneur.63.10.1469 schema:sameAs https://app.dimensions.ai/details/publication/pub.1047368769
287 rdf:type schema:CreativeWork
288 https://doi.org/10.1001/archneur.63.10.1479 schema:sameAs https://app.dimensions.ai/details/publication/pub.1044242012
289 rdf:type schema:CreativeWork
290 https://doi.org/10.1002/iub.478 schema:sameAs https://app.dimensions.ai/details/publication/pub.1047203985
291 rdf:type schema:CreativeWork
292 https://doi.org/10.1002/mds.20781 schema:sameAs https://app.dimensions.ai/details/publication/pub.1039904839
293 rdf:type schema:CreativeWork
294 https://doi.org/10.1002/mds.21543 schema:sameAs https://app.dimensions.ai/details/publication/pub.1014132319
295 rdf:type schema:CreativeWork
296 https://doi.org/10.1002/mds.25126 schema:sameAs https://app.dimensions.ai/details/publication/pub.1009326820
297 rdf:type schema:CreativeWork
298 https://doi.org/10.1002/mpr.282 schema:sameAs https://app.dimensions.ai/details/publication/pub.1028514340
299 rdf:type schema:CreativeWork
300 https://doi.org/10.1016/0005-2736(90)90272-p schema:sameAs https://app.dimensions.ai/details/publication/pub.1018056533
301 rdf:type schema:CreativeWork
302 https://doi.org/10.1016/0005-2760(96)00067-7 schema:sameAs https://app.dimensions.ai/details/publication/pub.1052965762
303 rdf:type schema:CreativeWork
304 https://doi.org/10.1016/j.bcmd.2004.04.004 schema:sameAs https://app.dimensions.ai/details/publication/pub.1002331703
305 rdf:type schema:CreativeWork
306 https://doi.org/10.1016/s0022-510x(96)00231-6 schema:sameAs https://app.dimensions.ai/details/publication/pub.1044787153
307 rdf:type schema:CreativeWork
308 https://doi.org/10.1074/jbc.m112.344473 schema:sameAs https://app.dimensions.ai/details/publication/pub.1012734720
309 rdf:type schema:CreativeWork
310 https://doi.org/10.1097/mpg.0b013e3180320667 schema:sameAs https://app.dimensions.ai/details/publication/pub.1028955820
311 rdf:type schema:CreativeWork
312 https://doi.org/10.1111/j.1468-1331.2008.02060.x schema:sameAs https://app.dimensions.ai/details/publication/pub.1033349556
313 rdf:type schema:CreativeWork
314 https://doi.org/10.1111/j.1468-1331.2009.02600.x schema:sameAs https://app.dimensions.ai/details/publication/pub.1007007344
315 rdf:type schema:CreativeWork
316 https://doi.org/10.1111/j.1468-1331.2010.03203.x schema:sameAs https://app.dimensions.ai/details/publication/pub.1002417286
317 rdf:type schema:CreativeWork
318 https://doi.org/10.1111/j.1470-8744.1998.tb00505.x schema:sameAs https://app.dimensions.ai/details/publication/pub.1004789617
319 rdf:type schema:CreativeWork
320 https://doi.org/10.1136/adc.2006.094268 schema:sameAs https://app.dimensions.ai/details/publication/pub.1027065426
321 rdf:type schema:CreativeWork
322 https://doi.org/10.1177/0883073812441999 schema:sameAs https://app.dimensions.ai/details/publication/pub.1037362094
323 rdf:type schema:CreativeWork
324 https://doi.org/10.1212/wnl.0b013e318225ab2e schema:sameAs https://app.dimensions.ai/details/publication/pub.1064356304
325 rdf:type schema:CreativeWork
326 https://doi.org/10.1212/wnl.0b013e3182494d51 schema:sameAs https://app.dimensions.ai/details/publication/pub.1064356788
327 rdf:type schema:CreativeWork
328 https://doi.org/10.1212/wnl.54.7.1505 schema:sameAs https://app.dimensions.ai/details/publication/pub.1064378560
329 rdf:type schema:CreativeWork
330 https://www.grid.ac/institutes/grid.12711.34 schema:alternateName University of Urbino
331 schema:name Department of Biomolecular Sciences, Università di Urbino “Carlo Bo”, Via Saffi 2, Erydel S.p.A, Via Sasso, 61029, 61029, Urbino, Urbino, Italy, Italy
332 rdf:type schema:Organization
333 https://www.grid.ac/institutes/grid.419457.a schema:alternateName Istituto Dermopatico dell'Immacolata
334 schema:name Istituto Dermopatico Immacolata, Via Monti Creta, 104, 00167, Roma, Italy
335 rdf:type schema:Organization
336 https://www.grid.ac/institutes/grid.4708.b schema:alternateName University of Milan
337 schema:name Department of Pediatrics, Università di Milano, Fondazione IRCCS Ca’ Granda, via Commenda 9, 20122, Milano, Italy
338 rdf:type schema:Organization
339 https://www.grid.ac/institutes/grid.7637.5 schema:alternateName University of Brescia
340 schema:name School in Reproductive and Developmental Science, Università di Trieste and Università di Brescia, Brescia, Piazzale Spedali Civili, 1 25123, Brescia, Italy
341 rdf:type schema:Organization
342 https://www.grid.ac/institutes/grid.7841.a schema:alternateName Sapienza University of Rome
343 schema:name Department of Clinical and Molecular Medicine, Sapienza Università di Roma, Via di Grottarossa 1035, 00189, Roma, Italy
344 Department of Molecular Medicine, Sapienza Università di Roma, Viale dell’Università 37, 00186, Roma, Italy
345 Department of Pediatrics and Child Neurology and Psychiatry, Sapienza Università di Roma, via dei Sabelli 108, 00185, Roma, Italy
346 rdf:type schema:Organization
 




Preview window. Press ESC to close (or click here)


...