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PODCAST

CURE SYNGAP1 PODCAST aka SYNGAP10

Syngap Research Fund, 501(c)(3)

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 parent-led public charity in the US that strives to accelerate research into treatments for SYNGAP1 so that we can help our loved ones in a timeframe that matters. Learn more at

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All Episodes

9:58

1,871 #SYNGAP1 Census, COMBINEDBrain RR-RGND,...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/10/02

Friday, October 2, 2026 — Week 40CENSUS — 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 Wonderful Rare Research Roundtable on Genetic Neurodevelopmental Disorders. COMBINEDBrain is a gift to the rare neuro community — hug them when you see them.

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9:57

CAMP4 ASCEND Webinar + #SYNGAP1 Goes Global:...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/09/26

Saturday, September 26, 2026 — Week 39CAMP4 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 - CAMP4 + LATAM hybrid meeting: “Path to the Clinical Trial in Argentina.” - Remember how they got here: THE CONFERENCE. Community → relationships → trial readiness. - Recording:

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9:58

ASCEND Trial Webinar + #SYNGAP1 Warriors, Biobank,...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/09/21

Monday, September 21, 2026 — Week 39CAMP4 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:

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9:57

UK MHRA Approves CAMP4 ASCEND + USA Today +...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/09/11

Friday, September 11, 2026 — Week 37CAMP4 — 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

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9:56

USA Today, Synchrony Conf, #EEC2026, Portugal &...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/09/08

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. Diagnosis and future therapies aren't enough. Families need care, education and support today.

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9:57

#Angelman ASO Fails, #AlexanderDisease ASO Wins:...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/09/06

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 — 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

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9:45

Big Week for #SYNGAP1: Trials, Data, #Epilepsy, Palo...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/08/31

Sunday, August 30, 2026 — Week 36TRIALS & 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 cash runway through the end of 2028. Q2 earnings / corporate

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9:58

#AdamsCamp & Tony in Denver: Support, Progress & Open...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/08/30

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 behavioral therapy. SYNGAP1 Connection at Camp! This was Tony’s fifth consecutive year at https://adamscamp.org/. That continuity made a difference. The Adam’s Camp team knew Tony

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9:57

Go Ada & Ford. Fundraise now. #CTRPoll Do it now....

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/08/10

Monday, August 10, 2026 - Week 33Make 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: (https://www.linkedin.com/posts/curesyngap1_syngap1census-syngap1-census-activity-7478070129340215296-6Hjx) CLINICAL TRIAL READINESS SURVEY - http://curesyngap1.org/CTRpoll Do it now, let us figure out the state of our community. CITIZEN Sign up now

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9:58

CAMP4 Trial Approved in Australia & Argentina,...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/08/07

Friday, August 7, 2026 - Week 32CAMP4 - 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: https://www.cnbc.com/2026/08/06/camp4-advances-rare-disease-treatment-for-syngap1-into-human-trials.html Where they mentioned approval in Argentina! $CAMP closed at $4.53 yesterday.

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9:52

#Future4Ford. 2Q26 #SYNGAP1Census = 1,806 Patients...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/07/05

#Future4Ford. 2Q26 #SYNGAP1Census = 1,806 Patients (+45) $CAMP #ValproateDemandBetter #S10e212Saturday, 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 - 1,806 https://curesyngap1.org/blog/syngap1-census-2026-update-45-q2-total-1806/ Per

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9:58

We need a #SYNGAP1 mouse lab? #BIO2026 #MDBR 19 days...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/06/26

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 left! New t-shirts! Buy for everyone! There are some funny ones this year and the classics. cureSYNGAP1.org/Bonfire GENETIC TESTING Valuable resource as you have questions

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9:57

#ILoveSomeoneWithSYNGAP1. June 21 is SYNGAP1...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/06/20

Friday, June 19, 2026 - Week 25June 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 https://curesyngap1.org/podcasts/syngap10/tony-update-thisisourstolose-more-great-results-from-camp4-nightofimpact-in-9-days-s10e208/ New family email, very good, lots of questions. It’s all here. Reach out, we are here for you and you are welcome in this

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9:56

#NightOfImpact a huge success & we are lucky to have...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/06/11

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, Helen Willsey & Me. Only got a video of John, which was a mistake. If you took one, please share. Here is John:

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9:57

Tony update. #ThisIsOursToLose. More great results...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/05/19

Tuesday, May 19, 2026 - Week 21Personal 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 & Progress - 1.1k views in 11 days. cureSYNGAP1.org/Burden https://www.linkedin.com/posts/syngap1-curesyngap1-raredisease-share-7458298251893448706-jfWF Send IMPACT REPORT

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9:53

Earnings reports: CAMP4 files. Stoke optimizing. ...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/05/08

Friday, May 8, 2026 - Week 19CURE 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 https://investors.camp4tx.com/news-releases/news-release-details/camp4-reports-first-quarter-2026-financial-results-and-corporate We submitted our first regulatory filing for CMP-002 in Australia

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9:57

Longitudinal Data Matters! Prioritized list of...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/05/06

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 families, while you are registering, please make sure to go to this one with Dr. Buxbaum from Mt. Sinai. curesyngap1.org/seaver

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9:56

Comment on Haystack Petition to FDA. New Board of...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/04/21

Monday, April 20, 2026 - Week 17CURE 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: https://static1.squarespace.com/static/5966cc2220099e91326caaec/t/69d7e016d5986e16ff09b64c/1775755288463/Letter+Head+Petition+for+rulemaking+to+amend%C2%A021+CFR+%C2%A7+314.126+and+%C2%A0%C2%A7312.47+%281%29.pdf Reuters 4/1:

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9:50

Global Advocacy. #NL51. Snippets! #SYNGAPcensus =...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/04/14

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: https://www.linkedin.com/posts/graglia_raredisease-activity-7448087944466259970-MADW?utm_source=share *** Dr. Lal’s post: ***

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9:57

DSC-III and Data - CURE SYNGAP1 is working tirelessly...

CURE SYNGAP1 PODCAST aka SYNGAP10 ·

2026/04/04

Friday, April 3, 2026 - Week 14Just 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 https://curesyngap1.org/podcasts/syngap10/dsc-rdcrn-ncats-nih-press-aav-in-cell-srf-at-cb-scramble-for-syngap1-s10e184/ We will have sites at SYNGAP1 established doctors, Dr. Wiltrout at Boston Children's Hospital/Harvard, Dr. Holder at Baylor and our very own

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222 results

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