Pediatric & Juvenile ALS
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Ulefnersen Early Access Program for FUS-ALS
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September 2026
Phase 3 clinical trial completed for FUS-ALS. Meets primary endpoint. Groundbreaking results!
Otsuka's Early Access Program for people living with FUS-ALS
April 2021
A Review of Gene Therapy for ALS.
January 2021
n-lorem Foundation to charitably provide experimental ASOs to treat patients with ultra-rare mutations.
April 2020
FUS Mutations Cause Most Cases of ALS in Adolescents.
