Neurodegenerative disease stories

Sep 21, 2026

FDA approves IntraBio's AQNEURSA as first treatment for ataxia-telangiectasia

The U.S. FDA has approved AQNEURSA (levacetylleucine) as the first and only approved treatment for ataxia-telangiectasia (A-T) in the United States or anywhere in the world. This marks the second FDA-approved indication for AQNEURSA.

Sep 21, 20262 sources

Sep 17, 2026

FDA approves Ultragenyx's Fayuvi, first gene therapy for Sanfilippo syndrome type A

The FDA granted full approval to Ultragenyx's gene therapy Fayuvi for Sanfilippo syndrome type A, marking the first-ever approved treatment for the ultra-rare, fatal neurodegenerative disease. The approval comes after a previous regulatory rejection and represents a significant milestone for patients with this devastating condition.

Sep 17, 20264 sources

Top claims

▪Brad Margus, founder of the A-T Children's Project, described the FDA approval of AQNEURSA as a historic day for the ataxia-telangiectasia community who have coped for decades without an approved treatment.
▪Ataxia-telangiectasia affects an estimated 1 in 40,000 people and is characterized by progressive cerebellar degeneration resulting in worsening ataxia that affects gait, balance, speech, and coordination.
▪IntraBio Inc. offers the AQNEURSA Cares support program to provide financial assistance and educational resources to help eligible patients navigate insurance coverage and access the treatment.

Subtopics

Drug Approval and Clinical Trials2 FDA drug approval2 Biotechnology1 Gene therapy1 Rare diseases1

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