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Efficacy and safety of arimoclomol in Niemann-Pick disease type C: Results from a double-blind, randomised, placebo-controlled, multinational phase 2/3 trial of a novel treatment.
Mengel, Eugen; Patterson, Marc C; Da Riol, Rosalia M; Del Toro, Mireia; Deodato, Federica; Gautschi, Matthias; Grunewald, Stephanie; Grønborg, Sabine; Harmatz, Paul; Héron, Bénédicte; Maier, Esther M; Roubertie, Agathe; Santra, Saikat; Tylki-Szymanska, Anna; Day, Simon; Andreasen, Anne Katrine; Geist, Marie Aavang; Havnsøe Torp Petersen, Nikolaj; Ingemann, Linda; Hansen, Thomas; Blaettler, Thomas; Kirkegaard, Thomas; Í Dali, Christine.
Afiliação
  • Mengel E; SphinCS GmbH, Institute of Clinical Science for LSD, Hochheim, Germany.
  • Patterson MC; Departments of Neurology, Pediatrics and Medical Genetics, Mayo Clinic, Rochester, Minnesota, USA.
  • Da Riol RM; Regional Coordination Center for Rare Diseases, Academic Hospital 'Santa Maria della Misericordia', Udine, Italy.
  • Del Toro M; Pediatric Neurology Department, Vall d'Hebron University Hospital, Barcelona, Spain.
  • Deodato F; Division of Metabolism, Ospedale Pediatrico Bambino Gesù, IRCCS, Rome, Italy.
  • Gautschi M; Department of Paediatrics, Division of Endocrinology, Diabetology and Metabolism, and Institute of Clinical Chemistry, Inselspital, University Hospital Bern, University of Bern, Bern, Switzerland.
  • Grunewald S; Department of Metabolic Medicine, Great Ormond Street Hospital, Institute of Child Health, UCL, NIHR Biomedical Research Center, London, UK.
  • Grønborg S; Centre for Inherited Metabolic Diseases, Copenhagen University Hospital (Rigshospitalet), Copenhagen, Denmark.
  • Harmatz P; Gastroenterology and Hepatology, UCSF Benioff Children's Hospital Oakland, Oakland, California, USA.
  • Héron B; Department of Pediatric Neurology, Reference Centre for Lysosomal Diseases, University Hospital Armand Trousseau, Paris, France.
  • Maier EM; Department of Inborn Errors of Metabolism, University of Munich Children's Hospital, Munich, Germany.
  • Roubertie A; Department of Neuropediatrics, Centre Hospitalier Universitaire de Montpellier, Montpellier, France.
  • Santra S; Department of Inherited Metabolic Disorders, Birmingham Children's Hospital, Birmingham, UK.
  • Tylki-Szymanska A; Department of Paediatrics, Nutrition and Metabolic Diseases, The Children's Memorial Institute, Warsaw, Poland.
  • Day S; Biostatistics, Clinical Trials Consulting & Training Limited, Buckingham, UK.
  • Andreasen AK; Orphazyme A/S, Copenhagen, Denmark.
  • Geist MA; Orphazyme A/S, Copenhagen, Denmark.
  • Havnsøe Torp Petersen N; Orphazyme A/S, Copenhagen, Denmark.
  • Ingemann L; Orphazyme A/S, Copenhagen, Denmark.
  • Hansen T; Orphazyme A/S, Copenhagen, Denmark.
  • Blaettler T; Orphazyme A/S, Copenhagen, Denmark.
  • Kirkegaard T; Orphazyme A/S, Copenhagen, Denmark.
  • Í Dali C; Orphazyme A/S, Copenhagen, Denmark.
J Inherit Metab Dis ; 44(6): 1463-1480, 2021 11.
Article em En | MEDLINE | ID: mdl-34418116
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Contexto em Saúde: 1_ASSA2030 Problema de saúde: 1_doencas_nao_transmissiveis Assunto principal: Doença de Niemann-Pick Tipo C / Hidroxilaminas Tipo de estudo: Clinical_trials / Observational_studies / Prognostic_studies Limite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Male Idioma: En Revista: J Inherit Metab Dis Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Alemanha
Texto completo
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XML
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Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Contexto em Saúde: 1_ASSA2030 Problema de saúde: 1_doencas_nao_transmissiveis Assunto principal: Doença de Niemann-Pick Tipo C / Hidroxilaminas Tipo de estudo: Clinical_trials / Observational_studies / Prognostic_studies Limite: Adolescent / Adult / Child / Child, preschool / Female / Humans / Male Idioma: En Revista: J Inherit Metab Dis Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Alemanha