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Over 1,600 families are caring for a loved one with the rare disease ”SynGAP” resulting from a variant of the SYNGAP1 gene. This weekly podcast is for them. -A quick summary of the latest news in the space. The host is Mike Graglia, co-founder & managing director of CURE SYNGAP1. CURE SYNGAP1 is a…
Tuesday, October 20, 2026 — Week 43 WHAT YOU CAN DO RIGHT NOW DAY-C remote developmental study — ages 9–25. Especially important for families who cannot participate in ProMMiS because travel/behavior makes in-person testing hard. ENDD@chop.edu…
Friday, October 2, 2026 — Week 40 CENSUS — 1,871 AND GROWING Q3 census: 1,871 known patients, +65 this quarter. First patient in Rwanda. USA +11, China +16, Brazil +6, France +4. Finding patients matters more than ever as trials approach. https://cureSYNGAP1.org/Census COMBINEDBRAIN — RARE RESEARCH ROUNDTABLE…
Saturday, September 26, 2026 — Week 39 CAMP4 ASCEND — WEBINAR #131 - SYNGAP1 ASCEND Phase 1/2 Trial: What You Need To Know. - Monday, Sept. 28 — 12 PM PT / 3 PM ET / 19:00 UTC. - Families: come hear directly from CAMP4 + ask your questions. https://cureSYNGAP1.org/AscendCT ARGENTINA — PATH TO THE CLINICAL TRIAL…
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…
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…
Tuesday, September 8, 2026 — Week 37 DON’T MISS ASO UPDATE Episode 217 came out over the weekend. Don't miss it. There was lots of ASO news in there. https://www.linkedin.com/feed/update/urn:li:activity:7502544874399928322/ NATIONAL PRESS USA TODAY Special-needs care / education crisis — and a SYNGAP1 family featured…
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. https://curesyngap1.org/podcasts/syngap10 ALEXANDER DISEASE On September 3, the FDA approved ZANVASTRO…
Sunday, August 30, 2026 — Week 36 TRIALS & DATA — CAMP4, RARE-X + ProMMiS CAMP4 / CMP-002 Australia and Argentina cleared. EU and UK filings underway. First-in-human Phase 1/2 study still targeted for Q4 2026. CMP-002 is designed to increase expression of the healthy SYNGAP1 copy. CAMP4 raised another $50M and reports…
Tony with us for a week after five months away… Way less screen addicted More independent Chubby - Less exercise, worse food Depressed Adam’s Camp was great as usual The support model was striking: the camper-to-therapist ratio was close to one-to-one, with skilled professionals in speech, occupational, physical, and…
Monday, August 10, 2026 - Week 33 Make sure to watch #S10e213 first! https://www.linkedin.com/posts/curesyngap1_camp4-trial-approved-in-australia-argentina-activity-7491598254027984896-wWTg 145 done! Out of 516 in US per last census…
Friday, August 7, 2026 - Week 32 CAMP4 - Gaining momentum - Approval for trials in Australia & +$50M of cash from investors:https://investors.camp4tx.com/news-releases/news-release-details/camp4-therapeutics-secures-australian-regulatory-clearance - Profile on CNBC Cures…
#Future4Ford. 2Q26 #SYNGAP1Census = 1,806 Patients (+45) $CAMP #ValproateDemandBetter #S10e212 Saturday, July 4, 2026 - Week 27 #Future4Ford! Already at $3,500+! Share. Thank you to the Family. This is the model for newly diagnosed, call us, set up a page, help us help your loved one. cureSYNGAP1.org/4Ford CENSUS…
Friday, June 26, 2026 - Week 26 BIO was good. Need a mouse lab to test molecules all year long. https://www.linkedin.com/posts/graglia_syngap-syngap1-mousemodels-ugcPost-7476041484408901633-Zldd/ MDBR was big, not just Justin A.! Thank you Dr. McKee, Heather and others. cureSYNGAP1.org/MDBR26Recap MERCH - 19 days…
Friday, June 19, 2026 - Week 25 June 21 is SYNGAP1 Awareness Day, but we have made it SYNGAP1 Awareness month! Why 6/21? Because 6p21.32 Donate! cureSYNGAP1.org/Donate #ThisIsOursToLose & #BurdenHopeProgress watch #S10e208…
Wednesday, June 10, 2026 - Week 24 #NightOfImpact was 15 Days ago! Photos: https://jeaniehorton.pixieset.com/curesyngap1nightofimpact2026/ Impact: $800k+, of which $300 was our match. Industry: Multiple Academics & Clinicians: Stanford, Berkeley & UCSF. Cross-pollination is always good. Speakers: Ash, John, Kathryn…
Tuesday, May 19, 2026 - Week 21 Personal Update via Syngap Stories: Page: cureSYNGAP1.org/stories41 Post: https://www.linkedin.com/posts/syngap1stories-syngap1-curesyngap1-share-7462125026276777984-OVBr #ThisIsOursToLose - Three steps to engaging friends with CURE SYNGAP1. Share CURE SYNGAP1 Video - Burden, Hope &…
Friday, May 8, 2026 - Week 19 CURE SYNGAP1 STRATEGY WORKING Our strategy has always been to derisk SYNGAP1 so that industry will invest and bring their skill and capital to bear. We leverage donor dollars to maximize impact. Yesterday was a great example: EARNINGS REPORTS CAMP4 1Q26 Results: $99M…
Wednesday, May 6, 2026 - Week 19 Congrats to GETA, CURE SYNGAP1 Australia & CAMP4 for a great weekend. https://www.linkedin.com/posts/syngap1-epilepsy-ugcPost-7457791427486466048-j8SB WEBINARS! CURE-ID Webinar tomorrow! Register now. Thu May 7, 2026 1:30pm – 3pm (PDT) cureSYNGAP1.org/cureID Next week, especially NY…
Monday, April 20, 2026 - Week 17 CURE SYNGAP1 joins the Haystack Project in petitioning FDA for more clarity. PR: https://static1.squarespace.com/static/5966cc2220099e91326caaec/t/69d7dbb80155f46e144ae2e5/1775754168227/4.9.26+press+release+petition_vf.pdf Petition…
Tuesday, April 14, 2026 - Week 16 Census is 1,761 https://curesyngap1.org/blog/syngap1-census-2026-update-54-q1-total-1761 Trip to Texas for UT Arlington and Cook Children's Systems innovation with Dr. Lal and Dr. Perry My post…
Friday, April 3, 2026 - Week 14 Just back from the DSC-III Kickoff Meeting! As announced back in September 2025. Really strong group of clinicians. https://dsc.rarediseasesnetwork.org/patient-advocacy-groups More on DSC in #S10e184…
Thursday, March 26, 2026 - Week 13 Thanks for NL50 Ed! cureSYNGAP1.org/NL50, I listened to Episode 1 and, as it turns out, it was March 12, 2021 (five 5️⃣ years ago), and it’s evergreen. SEVEN THINGS YOU NEED TO DO TO BE READY FOR CLINICAL TRIALS Stay Connected to CURE SYNGAP1. Fill in the Connect Form…
Friday, March 6, 2026 - Week 10 WHAT DO WE NEED $ FOR? I talked in Episode 197 #S10e197 about scientific priorities, and in Episode 200 #S10e200 about areas of activity beyond science grants. All of this is what we need to fund. SPRINT FOR SYNGAP1 Sprint for SYNGAP is coming fast– 49 DAYS. Make a difference. Raise…
Wednesday, February 25, 2026 - Week 9 Thank you Virginie, Eric & Paulina for being in Cold DC right now with the Everylife Foundation! https://www.linkedin.com/posts/curesyngap1_raredc2026-syngap1-curesyngap1-activity-7432425642295586816-IVDQ NATURAL HISTORY STUDY Sign up for Citizen Health cureSYNGAP1.org/Citizen and…
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