rare disease

2 entries tagged rare disease. All writing.

SEPTEMBER 21, 2026 Alexander Disease, and the First Drug Approved to Treat It What is Alexander disease, and what did the FDA actually approve for it? AxD is an ultra-rare, usually fatal genetic disorder of the brain's astrocytes, caused by mutations in the GFAP gene. On September 3, 2026, the FDA approved Zanvastro (zilganersen), an RNA-targeted drug given by quarterly spinal injection, as the first treatment aimed at the disease itself rather than its symptoms. The trial behind it enrolled 49 randomized patients. 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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