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Long-term safety and efficacy of pegunigalsidase alfa: A multicenter 6-year study in adult patients with Fabry disease.
Hughes, Derralynn; Gonzalez, Derlis; Maegawa, Gustavo; Bernat, John A; Holida, Myrl; Giraldo, Pilar; Atta, Mohamed G; Chertkoff, Raul; Alon, Sari; Almon, Einat Brill; Rocco, Rossana; Goker-Alpan, Ozlem.
Afiliación
  • Hughes D; LSDU, Royal Free London NHS Foundation Trust and University College London, London, United Kingdom. Electronic address: derralynnhughes@nhs.net.
  • Gonzalez D; Department of Haematology, Instituto Privado de Hematologia e Investigación Clínica, Asunción, Paraguay.
  • Maegawa G; Department of Pediatrics, New York-Presbyterian Morgan Stanley Children's Hospital, Vagelos College of Physicians and Surgeons, Columbia University Irving Medical Center, New York, NY.
  • Bernat JA; Division of Medical Genetics and Genomics, Stead Family Department of Pediatrics, University of Iowa Hospitals and Clinics, Iowa City, IA.
  • Holida M; Division of Medical Genetics and Genomics, Stead Family Department of Pediatrics, University of Iowa Hospitals and Clinics, Iowa City, IA.
  • Giraldo P; Centro de Investigación Biomédica en Red de Enfermedades Raras, Hospital de Dia Quiron, Zaragoza, Spain.
  • Atta MG; Division of Nephrology, Johns Hopkins School of Medicine, Baltimore, MD.
  • Chertkoff R; Department of Product Development, Protalix Biotherapeutics, Carmiel, Israel.
  • Alon S; Department of Product Development, Protalix Biotherapeutics, Carmiel, Israel.
  • Almon EB; Department of Product Development, Protalix Biotherapeutics, Carmiel, Israel.
  • Rocco R; Chiesi Farmaceutici S.p.A., Parma, Italy.
  • Goker-Alpan O; Lysosomal & Rare Disorders Research & Treatment Center, Fairfax, VA.
Genet Med ; 25(12): 100968, 2023 Dec.
Article en En | MEDLINE | ID: mdl-37634127
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Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Enfermedad de Fabry Tipo de estudio: Clinical_trials Límite: Adult / Female / Humans / Male Idioma: En Revista: Genet Med Asunto de la revista: GENETICA MEDICA Año: 2023 Tipo del documento: Article
Texto completo
Imprimir
XML
PubMed Links
Buscar en Google

Texto completo: 1 Banco de datos: MEDLINE Asunto principal: Enfermedad de Fabry Tipo de estudio: Clinical_trials Límite: Adult / Female / Humans / Male Idioma: En Revista: Genet Med Asunto de la revista: GENETICA MEDICA Año: 2023 Tipo del documento: Article