Addressing a drug delivery controversy: Evaluation of the data for IT versus IV administration of hydroxypropyl-beta-cyclodextrin for the treatment of Niemann Pick disease type C patients

Molecular Genetics and Metabolism 2026

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Authors: Caroline A. Hastings, Thorsten Marquardt

DOI: 10.1016/j.ymgme.2025.109458

Cited by: 0

Fields of Study & Subjects

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