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Safety and efficacy of elapegademase in patients with adenosine deaminase deficiency: A multicenter, open‐label, single‐arm, phase 3, and postmarketing clinical study

INTRODUCTION: Adenosine deaminase (ADA) deficiency is an ultrarare inherited purine metabolism disorder characterized by severe combined immunodeficiency. Elapegademase‐lvlr is a new pegylated recombinant bovine ADA used in enzyme‐replacement therapy (ERT) for ADA deficiency. Therefore, replacement...

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Detalles Bibliográficos
Autores principales:	Onodera, Masafumi, Uchiyama, Toru, Ariga, Tadashi, Yamada, Masafumi, Miyamura, Takako, Arizono, Hironori, Morio, Tomohiro
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	John Wiley and Sons Inc. 2023
Materias:	Review Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10367445/ https://www.ncbi.nlm.nih.gov/pubmed/37506145 http://dx.doi.org/10.1002/iid3.917

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