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 disease-specific treatment milestone for ataxia-telangiectasia after decades without a dedicated therapy.
Key companies, including Grace Therapeutics with GTX-102 and Quince Therapeutics with eDSP, along with other players, are advancing their respective candidates to compete with IntraBio’s AQNEURSA.
Ataxia-telangiectasia is one of those conditions ...
