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Long term follow-up to evaluate the efficacy of miglustat treatment in Italian patients with Niemann-Pick disease type C.
Fecarotta, Simona; Romano, Alfonso; Della Casa, Roberto; Del Giudice, Ennio; Bruschini, Diana; Mansi, Giuseppina; Bembi, Bruno; Dardis, Andrea; Fiumara, Agata; Di Rocco, Maja; Uziel, Graziella; Ardissone, Anna; Roccatello, Dario; Alpa, Mirella; Bertini, Enrico; D'Amico, Adele; Dionisi-Vici, Carlo; Deodato, Federica; Caviglia, Stefania; Federico, Antonio; Palmeri, Silvia; Gabrielli, Orazio; Santoro, Lucia; Filla, Alessandro; Russo, Cinzia; Parenti, Giancarlo; Andria, Generoso.
Afiliação
  • Fecarotta S; Department of Translational Medicine-Section of Pediatrics, Federico II University, Naples, Italy. simona.fecarotta@gmail.com.
  • Romano A; Department of Translational Medicine-Section of Pediatrics, Federico II University, Naples, Italy. alromano@unina.it.
  • Della Casa R; Department of Translational Medicine-Section of Pediatrics, Federico II University, Naples, Italy. roberto.dellacasa@unina.it.
  • Del Giudice E; Department of Translational Medicine-Section of Pediatrics, Federico II University, Naples, Italy. endelgiu@unina.it.
  • Bruschini D; Department of Translational Medicine-Section of Pediatrics, Federico II University, Naples, Italy. diana.bruschini@libero.it.
  • Mansi G; Department of Translational Medicine-Section of Pediatrics, Federico II University, Naples, Italy. giumansi@unina.it.
  • Bembi B; Regional Coordinator Centre for Rare Diseases, University Hospital "Santa Maria della Misericordia", Udine, Italy. bembi.bruno@aoud.sanita.fvg.it.
  • Dardis A; Regional Coordinator Centre for Rare Diseases, University Hospital "Santa Maria della Misericordia", Udine, Italy. dardis.andrea@aoud.sanita.fvg.it.
  • Fiumara A; Department of Pediatrics, Regional Referral Center for Inherited Metabolic Disease, University of Catania, Catania, Italy. agafiu@virgilio.it.
  • Di Rocco M; Department of Pediatrics, Unit of Rare Diseases, Gaslini Institute, Genoa, Italy. majadirocco@ospedale-gaslini.ge.it.
  • Uziel G; Unit of Child Neurology, The Foundation "Carlo Besta" Neurological Institute (IRCCS), Milan, Italy. uziel@istituto-besta.it.
  • Ardissone A; Unit of Child Neurology, The Foundation "Carlo Besta" Neurological Institute (IRCCS), Milan, Italy. Anna.Ardissone@istituto-besta.it.
  • Roccatello D; Center of Research on Immunopathology and Rare Diseases (CMID), San Giovanni Bosco Hospital and University of Turin, Turin, Italy. dario.roccatello@unito.it.
  • Alpa M; Center of Research on Immunopathology and Rare Diseases (CMID), San Giovanni Bosco Hospital and University of Turin, Turin, Italy. mirella.alpa@virgilio.it.
  • Bertini E; Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Diseases, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy. bertini@opbg.net.
  • D'Amico A; Department of Neurosciences, Unit of Neuromuscular and Neurodegenerative Diseases, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy. adeledamico@hotmail.com.
  • Dionisi-Vici C; Department of Pediatric Medicine, Division of Metabolism, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy. dionisi@opbg.net.
  • Deodato F; Department of Pediatric Medicine, Division of Metabolism, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy. deodato@opbg.net.
  • Caviglia S; Department of Neurosciences, Psychology Clinic Unit, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy. stefania.caviglia@opbg.net.
  • Federico A; Department of Medical, Surgical and Neurological Sciences, University of Siena, Siena, Italy. federico@unisi.it.
  • Palmeri S; Department of Medical, Surgical and Neurological Sciences, University of Siena, Siena, Italy. silvia.palmeri@unisi.it.
  • Gabrielli O; Department of Clinical Sciences, Polytechnic University of Marche, Ospedali Riuniti, Ancona, Italy. o.gabrielli@mercurio.it.
  • Santoro L; Department of Clinical Sciences, Polytechnic University of Marche, Ospedali Riuniti, Ancona, Italy. dott.luciasantoro@gmail.com.
  • Filla A; Department of Neurosciences, Reproductive and Odontostomatological Sciences, Federico II University, Naples, Italy. alessandro.filla@unina.it.
  • Russo C; Department of Neurosciences, Reproductive and Odontostomatological Sciences, Federico II University, Naples, Italy. cinziavaleria@hotmail.it.
  • Parenti G; Department of Translational Medicine-Section of Pediatrics, Federico II University, Naples, Italy. parenti@unina.it.
  • Andria G; Department of Translational Medicine-Section of Pediatrics, Federico II University, Naples, Italy. andria@unina.it.
Orphanet J Rare Dis ; 10: 22, 2015 Feb 27.
Article em En | MEDLINE | ID: mdl-25888393
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Texto completo: 1 Base de dados: MEDLINE Métodos Terapêuticos e Terapias MTCI: Terapias_biologicas / Aromoterapia Assunto principal: 1-Desoxinojirimicina / Inibidores Enzimáticos / Doença de Niemann-Pick Tipo C Tipo de estudo: Clinical_trials País/Região como assunto: Europa Idioma: En Revista: Orphanet J Rare Dis Ano de publicação: 2015 Tipo de documento: Article País de afiliação: Itália
Texto completo
Adicionar na Minha BVS
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Base de dados: MEDLINE Métodos Terapêuticos e Terapias MTCI: Terapias_biologicas / Aromoterapia Assunto principal: 1-Desoxinojirimicina / Inibidores Enzimáticos / Doença de Niemann-Pick Tipo C Tipo de estudo: Clinical_trials País/Região como assunto: Europa Idioma: En Revista: Orphanet J Rare Dis Ano de publicação: 2015 Tipo de documento: Article País de afiliação: Itália