Substantial survival benefit with adrabetadex treatment in individuals with infantile-onset Niemann-Pick disease type C (NPC)

Molecular Genetics and Metabolism 2026

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Authors: Elizabeth Berry‐Kravis, Laurence H. Keller, Lixia Jiao, John P. Winnike, Alexander M. Gold, Jason Camm, Forbes D. Porter

DOI: 10.1016/j.ymgme.2025.109341

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Glycogen Storage Diseases and Myoclonus Biomedical Research and Pathophysiology