Health Tip on New Treatment that Shows Promise in Niemann-Pick

Health tip that describes how cyclodextrin, a new treatment offers hope to patients with Niemann-Pick Type C disorder.

Liposomal formulations for treating lysosomal storage disorders

LifeArc publishes expert advice to support work around repurposing

Therapeutic advantages of combined gene/cell therapy strategies in

Pick's disease: Symptoms, causes, treatment, and more

Frontiers Epigenetic Approaches to the Treatment of Dental Pulp

NCATS Research Team Identifies Possible Treatment for Niemann-Pick

Lysosomal storage diseases: Diagnostic confirmation and management

Liposomal formulations for treating lysosomal storage disorders

Navigating the Rare Disease Space: Insights from Orphazyme's CEO

Investigational New Drug (IND) - The Addi and Cassi Fund

An image-based screen for chaperone drugs that correct mis

Niemann–Pick disease type C1 (NPC1) lysosomal lipid storage