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Long-term Follow-up of Cipaglucosidase Alfa/Miglustat in Ambulatory Patients with Pompe Disease: An Open-label Phase I/II Study (ATB200-02)

Recurrent Transient Global Amnesia with Contralateral Hippocampal Restricted Diffusion MRI Changes: A Case Report

EMBARK, a Phase 3 Trial Evaluating Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Duchenne Muscular Dystrophy (DMD): Study Design and Baseline Characteristics