Long-term safety and effectiveness of arimoclomol in adult and pediatric Niemann-Pick disease type C patients in the US Early Access Program (EAP)

Molecular Genetics and Metabolism 2026

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Authors: Damara N. Ortiz, Nicolas J. Abreu, Walla Al‐Hertani, Elizabeth Berry‐Kravis, Radhika Dhamija, Can H. Ficicioglu, Caroline A. Hastings, Paul Hillman, Kristina Julich, Nancy Leslie, Melinda M. Peters, Paula Schleifer
+3 more Ronan J. O'Reilly, Blair Orr, Christine í Dali
DOI: 10.1016/j.ymgme.2025.109571
Cited by: 0

Fields of Study & Subjects

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

Publication details

Journal / source
Molecular Genetics and Metabolism
Volume
147
Issue
2
Pages
109571–109571
Publication date
1 February 2026
Language
EN