ASCEND Trial Webinar + #SYNGAP1 Warriors, Biobank, HEOR & Industry News #S10E220

Monday, September 21, 2026 — Week 39 CAMP4 ASCEND TRIAL — WEBINAR #131 CAMP4’s SYNGAP1 ASCEND Phase 1/2 Trial — What You Need To Know. Tuesday, Sept. 29 — 12 PM ET / 9 AM PT. REGISTER https://curesyngap1.org/calendar/webinar-131-camp4-syngap1-ascend-phase-1-2-trial/ SCRAMBLE FOR SYNGAP — 12 DAYS 5th Annual — Oct. 3, Greer, SC. Julie + family back on WSPA 7 News — including some very real family moments. SOLD OUT. Five years of building awareness + community. https://youtu.be/GcD58deUPtU Event: https://cureSYNGAP1.org/Scramble WARRIORS — KEEP TELLING THE STORIES NEW: Weston, 22 — diagnosed only 6 months ago. Late diagnosis! https://cureSYNGAP1.org/Warrior Will was Warrior #21 at age 9. He’s now 16. https://cureSYNGAP1.org/Will Kiera — Warrior #172 — now 6. https://cureSYNGAP1.org/Kiera These stories matter. Keep sharing them. COMBINEDBrain BIOBANK — ONLY 2 LOCATIONS LEFT Boston — Oct. 8–9. Denver — Dec. 3–4 at the CURE SYNGAP1 Conference. Email Lauren@cureSYNGAP1.org. HEOR — DOCUMENTING THE REAL BURDEN Building our Health Economics & Outcomes Research work with payers & clinicians in mind. Anxiety + behavior management matter.  e.g. Rett: 323 caregivers — https://pubmed.ncbi.nlm.nih.gov/42570201/ e.g. Angelman: U.S. study of 105 caregivers estimated average annual caregiving economic impact at $79,837 — 53% from employment impacts/lost productivity. https://pubmed.ncbi.nlm.nih.gov/39985061/ HELP WANTED — PRESS We have a backlog of CURE SYNGAP1 press releases. Know PR / communications? We need help. Volunteer skills matter just as much as fundraising. IONIS — ANGELMAN Ionis published a community letter after the GTX-102/ASPIRE results. Relevant follow-up to my discussion in E217. Read it directly — 2’MOE https://assets.ctfassets.net/qj7dcdpo5rmb/nBQEr7TJQIyexzQu229Dl/a5ae4fff28861385532da0c8390b1c30/PA-US-AS-260005_-_Community_Statement_on_Status_of_Obudanersen_Program_-_FINAL_-_Sept_2026.pdf ULTRAGENYX — CONGRATULATIONS FDA FULL APPROVAL for FAYUVI / UX111 for Sanfilippo syndrome Type A. First-ever FDA-approved treatment for MPS IIIA. Single-dose IV AAV9 gene therapy. Second approved gene therapy for RARE. https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-approval-fayuvitm-gene-therapy-first-ever UPCOMING SHOOT FOR SYNGAP1 — Nov. 14, Hurricane, UT https://cureSYNGAP1.org/Shoot FIGHT FOR FELIPE — Nov. 28, Boston https://cureSYNGAP1.org/Fight CURE SYNGAP1 CONFERENCE — Dec. 3–4, Denver https://cureSYNGAP1.org/Reg26 PUBMED 50 SYNGAP1 publications in 2026 — +11 vs. Week 39. https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date USA: Use your ICD-10, F78.A1 SOCIAL MATTERS 5,268 LinkedIn — +28 since E219. https://www.linkedin.com/company/curesyngap1 1.62k Yo