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.

Consensus clinical management guidelines for Niemann-Pick disease

Moving away from one disease at a time: Screening, trial design

Finding a Breakthrough for Tucker and the World - The Children's

Repurposing the Ebola and Marburg Virus Inhibitors Tilorone

Regenerative medicine: prenatal approaches - The Lancet Child

2023 Guideline for the Management of Patients With Aneurysmal

Niemann-Pick disease: Causes, symptoms, treatment, and more

2023 Guideline for the Management of Patients With Aneurysmal

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

Recommendations for the diagnosis and management of Niemann–Pick