rare disease

1 entry tagged rare disease. All writing.

SEPTEMBER 19, 2026 The First Approved Treatment for Ataxia in Ataxia-Telangiectasia What is ataxia-telangiectasia, and does the drug the FDA just approved for it actually work? A-T is a rare, inherited disorder in a DNA-repair gene that causes progressive loss of coordination starting in early childhood. On September 18, 2026, the FDA approved levacetylleucine (Aqneursa) as the first treatment for the ataxia itself, based on a 73-patient trial showing a real, statistically significant improvement on the standard ataxia rating scale.

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