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

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

Abstract 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, repl...

Full description

Bibliographic Details
Main Authors: Masafumi Onodera, Toru Uchiyama, Tadashi Ariga, Masafumi Yamada, Takako Miyamura, Hironori Arizono, Tomohiro Morio
Format: Article
Language:English
Published: Wiley 2023-07-01
Series:Immunity, Inflammation and Disease
Subjects:
adenosine deaminase deficiency
elapegademase
enzyme replacement therapy
immunologic deficiency syndromes
Online Access:https://doi.org/10.1002/iid3.917

Internet

https://doi.org/10.1002/iid3.917

Similar Items