Health Tip on New Treatment that Shows Promise in Niemann-Pick Type C Disorder - Health Tips

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

Emptying the stores: lysosomal diseases and therapeutic strategies

What You See and What You Don't: Diagnosing Niemann Pick, Type C

Diagnosis and management of progressive ataxia in adults

Discovery of a Small-Molecule Modulator of the Autophagy-Lysosome

Recommendations for the diagnosis and management of Niemann–Pick

Rare Cancer Continuing Medical Education - National Organization

Health Tips Related to Pediatrics Speciality

Health Tips Related to Pediatrics Speciality

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

Gene therapy shows promise for treating Niemann-Pick disease type

LifeArc publishes expert advice to support work around repurposing

News from MetabERN about Networks - MetabERN

Frontiers Mesenchymal Stem Cell-Based Therapy for Lysosomal

IJMS, Free Full-Text

Precision medicine in monogenic inflammatory bowel disease