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.

Potential therapeutic strategies for photoreceptor degeneration: the

Push to spur drugs for deadly rare diseases

2022 drug approvals: Biopharma delivered 34 new drugs

Combination therapy a potential strategy for treating Niemann-Pick

Frontiers Current and Future Prospects for Gene Therapy for Rare

Gene therapy shows promise for treating Niemann-Pick disease type

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

Experimental treatment for Niemann-Pick disease appears safe

Gene therapy for lysosomal storage diseases: the lessons and

Confronting The Challenge of Rare Diseases

An image-based screen for chaperone drugs that correct mis