Arimoclomol and levacetylleucine for the treatment of neurologic manifestations of Niemann-Pick disease type C: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)

Authors

Jorge L. Rodriguez-Gil, Division of Medical Genetics, Department of Pediatrics, Stanford University School of Medicine, Stanford, CA.
Wendy E. Smith, Division of Genetics, Department of Pediatrics, Maine-Health Maine Medical Center, Portland, ME.
Irene J. Chang, Division of Medical Genetics, Department of Pediatrics, University of California San Francisco, San Francisco, CA.
Bryce A. Mendelsohn, Department of Medical Genetics, Kaiser Permanente Oakland Medical Center, Oakland, CA.

Document Type

Article

Publication Date

12-23-2025

Institution/Department

Pediatrics

Journal Title

Genetics in medicine open

First Page

103462

Recommended Citation

Rodriguez-Gil, Jorge L.; Smith, Wendy E.; Chang, Irene J.; and Mendelsohn, Bryce A., "Arimoclomol and levacetylleucine for the treatment of neurologic manifestations of Niemann-Pick disease type C: A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)" (2025). MaineHealth Maine Medical Center. 4298.
https://knowledgeconnection.mainehealth.org/mmc/4298