Trappsol® Cyclo™ in infants and toddlers with Niemann-Pick disease type C1: Interim results from the open-label sub-study of the transport-NPC™ phase 3 trial

Molecular Genetics and Metabolism 2026

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Authors: Orna Staretz‐Chacham, Fatih Ezgu, Eugen Mengel, Sema Kalkan Uçar, Ronen Spiegel, Caroline A. Hastings

DOI: 10.1016/j.ymgme.2025.109624

Cited by: 0

Fields of Study & Subjects

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