UK MHRA Approves CAMP4 ASCEND + USA Today + Rare-X/Citizen Health + 6 Things #S10E219

Friday, September 11, 2026 — Week 37

CAMP4 — ASCEND EXPANDS TO THE UK

• UK MHRA authorizes UK sites in CAMP4's Phase 1/2 CMP-002 trial.
• UK joins Australia + Argentina. EU filing remains under review.
• First-in-human trial still targeted to begin Q4 2026.
• Another major step toward our first disease-modifying clinical trial.
  https://investors.camp4tx.com/news-releases/news-release-details/camp4-therapeutics-receives-authorization-united-kingdom-phase

CAMP4 ANALYST DAY — SEPT. 28

• 12–1:30 PM ET. Trial design + unmet need + early pipeline.
• CURE SYNGAP1 participating https://investors.camp4tx.com/news-events/events
• We will have a day after, webinar, stay tuned for details.

USA TODAY — SYNGAP1 IN PRINT

• SYNGAP1 family story appeared in USA TODAY's national print edition this week.
• Families need education, care + support TODAY.
  https://www.usatoday.com/story/life/health-wellness/2026/09/01/kids-special-education-school-shortage-care/90851980007/

RARE-X + CITIZEN HEALTH

• Global Genes selects Citizen Health technology to power RARE-X.
• Important for us: ProMMiS uses Rare-X; CURE SYNGAP1 already works with Citizen.
  https://www.prnewswire.com/news-releases/global-genes-partners-with-citizen-health-to-power-rare-x-302870871.html

SIX THINGS U.S. FAMILIES CAN DO

• Our Take Action page is LIVE.
• Don't just read it. Keep coming back until you've done all six.
  https://curesyngap1.org/TakeAction

IEP HELP — CITIZEN HEALTH

• Oct. 4: IEP deep dive + Q&A with Staci Zimmerman, M.Ed.
• Register https://curesyngap1.org/calendar/what-nobody-tells-you-about-ieps-citizen-health-webinar/

RESEARCH — HELP WANTED

• 2-year SYNGAP1 postdoc — Sapienza University of Rome.
• Patient iPSCs, cortical neurons + brain organoids.
• Deadline Sept. 30. Know someone? Amplify it. https://www.sins.it/job_offer/postdoctoral-position-available-at-sapienza-university-of-rome/ 

COMMUNITY QUICK HITS

• NEW WARRIOR: Felipe, age 3. Parents Brian + Alana organizing Fight for Felipe.
• Want to fundraise? New support form: https://cureSYNGAP1.org/Fundraise
• Café SYNGAP1 #40 + #41: GRIN2A + GRIN2B communities. https://cureSYNGAP1.org/Cafe
• Night of Impact recap live. Time to start planning the next one. https://cureSYNGAP1.org/SF26Recap

UPCOMING EVENTS — COUNTDOWN

SHOOT FOR SYNGAP1 — 64 DAYS
November 14 — Hurricane, Utah
Aiming for a Cure — Shooting for Hope https://cureSYNGAP1.org/Shoot

FIGHT FOR FELIPE — 78 DAYS
November 28 — Boston, Massachusetts https://cureSYNGAP1.org/Fight

CURE SYNGAP1 CONFERENCE — 83 DAYS
December 3–4 — Denver, Colorado https://cureSYNGAP1.org/Reg26

USA 🇺🇲: Use your ICD-10, F78.A1

SOCIAL MATTERS

5,240 LinkedIn — https://www.linkedin.com/company/curesyngap1

1.62k YouTube https://www.youtube.com/@CureSYNGAP1

43.7k Instagram https://www.instagram.com/curesyngap1

Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10

Episode 219 of #Syngap10 #SYNGAP1 #RareDisease #CureSYNGAP1 #PatientAdvocacy