AQNEURSA

Oct 02, 2026

Summary  Ataxia-telangiectasia is a rare, progressive genetic disorder affecting an estimated 1 in 40,000–100,000 people worldwide. AQNEURSA received FDA approval on September 18, 2026, becoming the first and only approved treatment for ataxia in patients with ataxia-telangiectasia and marking the first...

May 02, 2025

Niemann-Pick disease type C (NPC) is a rare, aggressive, and life-threatening neurovisceral disorder with a profound impact on both patients and the healthcare system. Caused by mutations in the NPC1 or NPC2 genes, NPC disrupts intracellular lipid transport, leading to toxic accumulation of cholesterol and other li...