Real-world safety and effectiveness of arimoclomol in patients with NPC: Outcomes from the US early access program (EAP) over a 4-year period

Elizabeth Berry‐Kravis; Nicolas J. Abreu; Walla Al‐Hertani; Radhika Dhamija; Can H. Ficicioglu; Kristina Julich; Caroline A. Hastings; Paul Hillman; Nancy Leslie; Damara N. Ortiz; Melinda Peters; Paula Schleifer; Ronan J. O'Reilly; Blair Orr; Christine í Dali

doi:10.1016/j.ymgme.2025.109339

Real-world safety and effectiveness of arimoclomol in patients with NPC: Outcomes from the US early access program (EAP) over a 4-year period

Molecular Genetics and Metabolism 2026

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Authors: Elizabeth Berry‐Kravis, Nicolas J. Abreu, Walla Al‐Hertani, Radhika Dhamija, Can H. Ficicioglu, Kristina Julich, Caroline A. Hastings, Paul Hillman, Nancy Leslie, Damara N. Ortiz, Melinda Peters, Paula Schleifer

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Ronan J. O'Reilly, Blair Orr, Christine í Dali

DOI: 10.1016/j.ymgme.2025.109339

Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Metabolism and Genetic Disorders Genetic Neurodegenerative Diseases

Publication details

Journal / source: Molecular Genetics and Metabolism
Volume: 147
Issue: 2
Pages: 109339–109339
Publication date: 1 February 2026
Language: EN