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 YouTube. https://www.youtube.com/@CureSYNGAP1

Like and subscribe wherever you listen. https://cureSYNGAP1.org/podcasts/syngap10

Episode 220 of #Syngap10 #RareDisease #CureSYNGAP1 #PatientAdvocacy