#Angelman ASO Fails, #AlexanderDisease ASO Wins: #SYNGAP1 CAMP4 ASO? NYT on Clinical Trials #S10E217 Sunday, September 6, 2026 — Week 36 🛑 If you're reading this on social media, these show notes are going to get cut off. Please go to the podcast homepage and click on episode 217 to get the full show notes. curesyngap1.org/podcasts/syngap10 ALEXANDER DISEASE On September 3, the FDA approved ZANVASTRO — zilganersen — from Ionis (IONS) for Alexander disease. It is the first FDA-approved treatment for Alexander disease and the first therapy to directly target the underlying protein buildup driving the disease. Alexander disease is caused by mutations in GFAP. Zilganersen reduces production of abnormal GFAP protein. fda.gov/news-events/press-announcements/fda-approves-first-drug-treat-alexander-disease ANGELMAN — ULTRAGENYX ASPIRE FAILS On September 2, Ultragenyx (RARE) announced results from its Phase 3 Aspire trial of apazunersen, formerly GTX-102, for Angelma...