CURE SYNGAP1 Podcast

Hosted by Mike Graglia, CURE SYNGAP1

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214

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Aug 2026

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The CURE SYNGAP1 Podcast is a weekly blog to keep parents and families up to date on what the team at CURE SYNGAP1 is doing to advocate for patients and advance research into SYNGAP1. If you do nothing else, listen to or watch this and let us know what you think. There are over 1,675 diagnosed patients on earth according to SRF's #SyngapCensus. If you are related to one of those patients or are a researcher or clinician interested in SYNGAP1, this podcast is for you. Learn more at https://cureSYNGAP1.org

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August 10, 2026Episode 2149 min

Go Ada & Ford. Fundraise now. #CTRPoll Do it now. Porter’s Sib Story. ORCA Study soon! #S10e214

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: ( 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! https://www.citizen.health/ai-advocate/syngap1 Everyone with a sick kid needs this. Different from CONNECT : https://curesyngap1.org/curesyngap1connect Make sure we know how to reach you. STUDIES ARE HAPPENING - Albus, thank you to those families. Plug it in.- Combi[o]mics - Thank you for doing this if invited.- Everyone will get an invite to do an ORCA Validation Study, please do this. If you don’t get that email by end of August, bug us. We are only inviting those we can confirm have a mutation. PERSONAL FUNDRAISERS Fundraisers advance our mission & help you connect to community #ForAda curesyngap1.org/forada is over $104k of $10k #4Ford curesyngap1.org/4ford is over $20k of $20k SIBLINGS - Go Porter! cureSYNGAP1.org/Sibling THINGS TO LOOK FORWARD TO… 5TH SCRAMBLE FOR SYNGAP, SC – 54 days till October 3rd Classic case of a small event becoming an institution! cureSYNGAP1.org/Scramble26 SHOOT FOR SYNGAP, UT – 96 days till November 14th curesyngap1.org/calendar/shoot-for-syngap1 FIGHT FOR FELIPE, MA - 111 days till November 29th curesyngap1.org/Fight26 CURE SYNGAP1 CONFERENCE - 115 days until December 3rd & 4th cureSYNGAP1.org/Reg26 rooms available: ‭ cureSYNGAP1.org/denhyatt for $159. In on Wed, out on Saturday. PUBMED Pubmed 2026 is at 47 . +14 vs the week. (61 last year was +9) We are already at the 3rd highest year. https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date USA 🇺🇲: Use your ICD-10, F78.A1 SOCIAL MATTERS 5,159 LinkedIn. https://www.linkedin.com/company/curesyngap1 1.6k YouTube. https://www.youtube.com/@CureSYNGAP1 11.1k Twitter https://twitter.com/cureSYNGAP1 43.7k Insta https://www.instagram.com/curesyngap1 Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10 Episode 214 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

August 7, 2026Episode 2139 min

CAMP4 Trial Approved in Australia & Argentina, Connect for news, Ada & Ford Fund a grant, #S10e213

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: 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. https://www.google.com/finance/beta/quote/CAMP:NASDAQ MAKE SURE YOU ARE REGISTERED WITH CONNECT https://curesyngap1.org/curesyngap1connect/ USA 🇺🇲: Use your ICD-10, F78.A1 CITIZEN Sign up now! https://www.citizen.health/ai-advocate/syngap1 SOCIAL MATTERS 5,156 LinkedIn. https://www.linkedin.com/company/curesyngap1 1.6k YouTube. https://www.youtube.com/@CureSYNGAP1 11.1k Twitter https://twitter.com/cureSYNGAP1 43.7k Insta https://www.instagram.com/curesyngap1 Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10 Episode 213 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

July 5, 2026Episode 2129 min

#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 - 1,806 https://curesyngap1.org/blog/syngap1-census-2026-update-45-q2-total-1806/ Per https://docs.google.com/spreadsheets/d/1oJwMysR2wyTxe91zLlKJglNa0NySPxkBF0PRiV6mBmM/edit?usp=sharing , no change in Germany, UK, or Netherlands. This is clearly wrong. MERCH - 9 days left! New t-shirts! Buy for everyone! There are some funny ones this year and the classics. cureSYNGAP1.org/Bonfire CAMP4 has been busy! Meeting with one of our families: https://www.linkedin.com/posts/syngap1-share-7473374060614201345-SeKO/ Shoutout on NASDAQ: https://www.linkedin.com/posts/camp4-therapeutics_syngap1awareness-regrna-syngap1-activity-7474823632972820480-UMqi $CAMP closed at $4.43 yesterday. https://www.google.com/finance/beta/quote/CAMP:NASDAQ BONES – This is not medical advice, I am not a doctor. Talk to a doctor AND know your facts . https://curesyngap1.org/blog/navigating-a-lifetime-of-diagnoses-michaels-syngap1-journey-and-the-effects-of-anti-seizure-medications-on-bone-density/ Valproate is the active Ion, it comes from Valproic Acid (Depakene), Sodium Valproate or Divalproex Sodium (Depakote). They all cause bone loss by stoping stomach from absorbing, then robbing bones, then flushing out. More and longer is worse. Vitamin D and Calcium can help but they need to start early and it’s better to just find another drug. Also insurance likes it because it’s cheap and old – demand better. “Valproic acid and clobazam were commonly used for epilepsy treatment, while risperidone, aripiprazole, and guanfacine were commonly used for behavior management. Valproate and lamotrigine were more effective at reducing seizure frequencies or maintaining seizure freedom than other anti-seizure medications.” Clinical signatures of SYNGAP1-related disorders through data integration https://pmc.ncbi.nlm.nih.gov/articles/PMC12419475/ THINGS TO LOOK FORWARD TO… 5TH SCRAMBLE FOR SYNGAP, SC – 91 days till October 3rd Classic case of a small event becoming an institution! cureSYNGAP1.org/Scramble26 SHOOT FOR SYNGAP, UT – 133 days till November 14th curesyngap1.org/calendar/shoot-for-syngap1/ FIGHT FOR FELIPE, MA - 148 days till November 29th curesyngap1.org/Fight26 CURE SYNGAP1 CONFERENCE - 152 days until December 3rd & 4th cureSYNGAP1.org/Denver rooms available: ‭ cureSYNGAP1.org/denhyatt for $159. In on Wed, out on Saturday. PUBMED Pubmed 2026 is at 40 . +13 vs the week. (61 last year was +9) We are already at the 4th highest year. https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date Thanks to Dr. Stephen Smith for this exciting paper, especially in the context of ASOs…Genetic rescue of disrupted synaptic protein interaction network dynamics following SYNGAP1 reactivation https://pubmed.ncbi.nlm.nih.gov/42362191/ USA 🇺🇲: Use your ICD-10, F78.A1 SOCIAL MATTERS 5,106 LinkedIn. https://www.linkedin.com/company/curesyngap1 1.59k YouTube. https://www.youtube.com/@CureSYNGAP1 11.1k Twitter https://twitter.com/cureSYNGAP1 43.7k Insta https://www.instagram.com/curesyngap1 Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10 Episode 212 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

June 29, 2026Episode 2119 min

We need a #SYNGAP1 mouse lab? #BIO2026 #MDBR 19 days for Merch. #GeneticTesting #ASOwebinar #S10e211

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. curesyngap1.org/GT WARRIORS - Phoenix and Kenna cureSYNGAP1.org/Warrior THINGS TO LOOK FORWARD TO… WEBINAR: Understanding ASOs & Informed Consent June 30 4:30 ET curesyngap1.org/calendar/understanding-asos-informed-consent 5TH SCRAMBLE FOR SYNGAP, SC – 99 days till October 3rd Classic case of a small event becoming an institution! cureSYNGAP1.org/Scramble26 SHOOT FOR SYNGAP, UT – 141 days till November 14th fb.watch/HRnwf9FnEB FIGHT FOR FELIPE, MA - 155 days till November 29th curesyngap1.org/Fight26 CURE SYNGAP1 CONFERENCE - 159 days until December 3rd & 4th cureSYNGAP1.org/Denver rooms available: ‭ cureSYNGAP1.org/denhyatt for $159. In on Wed, out on Saturday. PUBMED Pubmed 2026 is at 38 . +12 vs the week. (61 last year was +9) We are already at the 4th highest year. https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date Props to Dr. Frazier for yet another paper on SYNGAP1 based on his study, one of the best grants ever. https://onlinelibrary.wiley.com/doi/10.1111/dmcn.70337 USA 🇺🇲: Use your ICD-10, F78.A1 SOCIAL MATTERS 5,074 LinkedIn. https://www.linkedin.com/company/curesyngap1 1.59k YouTube. https://www.youtube.com/@CureSYNGAP1 11.1k Twitter https://twitter.com/cureSYNGAP1 43.8k Insta https://www.instagram.com/curesyngap1 $CAMP closed at $3.92 yesterday. https://www.google.com/finance/beta/quote/CAMP:NASDAQ Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10 Episode 211 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

June 20, 2026Episode 2109 min

#ILoveSomeoneWithSYNGAP1. June 21 is SYNGAP1 Awareness day. $159 rooms at the GrandHyatt! #S10e210

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 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 community – you are part of it whether you like it or not. Raise money. Join our host committee for 2nd SF event next year. Believe that we are doing the best we possibly can, and you would be adrift without a PAG. If you think this is painful, imagine if we weren't here! Register for the Conference. Book tickets to DEN now. cureSYNGAP1.org/Denver Join us -- make this org better for all of us and all our kids for all their lives. Complex work doesn't fit neatly in 10 minutes anymore... Health Economics and Market Access work is going well, we will put out an RFP shortly. NATURAL HISTORY Goes On! Make sure you are in it. https://curesyngap1.org/resources/studies/syngap1-prommis/ Orlando/McKee Grant "Validating Remote Developmental Assessments in SYNGAP1-Related Disorders" cureSYNGAP1.org/PR49 🤖 ARI Sign up now, s/he keeps getting better, and then connect to Citizen Health, if you are not on Citizen Health, fix that. 1. https://www.citizen.health/ai-advocate/syngap1 2. https://ari.citizenhealth.com/ KUDOS - The fifth MDBR, Justin Albrecht rode 70 remote in NC. Raised $2k. - Rachel Jasiczek (Yah-she-check) was a guest on Remarkable Futures Podcast - https://open.spotify.com/episode/7dYmHcXEu58H4C8VCoZ9V1?si=x3xvui86TY2cyGHyS3mGDQ - Collective: CURE SYNGAP1 Collective - cureSYNGAP1.org/PR48 Collective page - cureSYNGAP1.org/Collective More fundraisers! WARD - Shoot for SYNGAP on November 14th in Utah! https://fb.watch/HRnwf9FnEB/ Another in Boston in the works… WEBINAR: Understanding ASOs & Informed Consent June 30 4:30 ET https://curesyngap1.org/calendar/understanding-asos-informed-consent/ 5TH SCRAMBLE FOR SYNGAP, SC – 106 days till October 3rd Classic case of a small event becoming an institution! cureSYNGAP1.org/Scramble26 CURE SYNGAP1 CONFERENCE - 166 days until December 3rd cureSYNGAP1.org/Denver rooms available: ‭ cureSYNGAP1.org/denhyatt for $159. In on Wed, out on Saturday. USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142 PUBMED Pubmed 2026 is at 37 . +12 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date SOCIAL MATTERS 5,058 LinkedIn. https://www.linkedin.com/company/curesyngap1 1.58k YouTube. https://www.youtube.com/@CureSYNGAP1 11.1k Twitter https://twitter.com/cureSYNGAP1 45k Insta https://www.instagram.com/curesyngap1 $CAMP closed at $4.13 yesterday. https://www.google.com/finance/beta/quote/CAMP:NASDAQ Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10 Episode 210 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

June 11, 2026Episode 2099 min

#NightOfImpact a huge success & we are lucky to have Dr. Helen R Willsey. #S10e209

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: https://www.linkedin.com/posts/graglia_still-reflecting-on-our-inaugural-cure-syngap1-ugcPost-7467439971294048256-cUf9/ Dr. Willsey Rocks. Willsey Press Release https://www.eurekalert.org/news-releases/1130924 (both were at NoI). A few other points on HRW, as we call her. Simons: https://curesyngap1.org/blog/future-research-for-syngap1-how-helen-willsey-broke-new-ground-frogs-in-hand/ Willsey in Neuron 2021: https://www.cell.com/neuron/pdf/S0896-6273(21)00002-7.pdf (Frogs) Birtele in Nature Neuroscience 2023: https://www.nature.com/articles/s41593-023-01477-3 (Confirms) McCluskey in Nature Communications 2025: https://www.nature.com/articles/s41467-025-57342-3 (GI)Kostyanovskaya in BioRxiv 2025: https://pubmed.ncbi.nlm.nih.gov/39677731 (cilium) 5TH SCRAMBLE FOR SYNGAP, SC – 114 days Classic case of a small event becoming an institution! cureSYNGAP1.org/Scramble26 CURE SYNGAP1 CONFERENCE - 175 days cureSYNGAP1.org/Pre USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142 PUBMED Pubmed 2026 is at 35 . +11 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date SOCIAL MATTERS 5,045 LinkedIn. https://www.linkedin.com/company/curesyngap1 1.58k YouTube. https://www.youtube.com/@CureSYNGAP1 11.1k Twitter https://twitter.com/cureSYNGAP1 45k Insta https://www.instagram.com/curesyngap1 $CAMP closed at $4.34 today. https://www.google.com/finance/beta/quote/CAMP:NASDAQ Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10 Episode 209 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

May 19, 2026Episode 2089 min

Tony update. #ThisIsOursToLose. More great results from $CAMP4. #NightOfImpact in 9 days! #S10e208

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. 1. 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 2. Send IMPACT REPORT - Tool for family, newly diagnosed & Fundraising. cureSYNGAP1.org/Impact or cureSYNGAP1.org/Impact25 3. Ask to Donate curesyngap1.org/donate/ CAMP4 Joining us at the Night of Impact (with Stoke, Acadia, BioMarin, Gondola & others) and… https://www.biospace.com/press-releases/camp4-therapeutics-to-present-new-preclinical-data-demonstrating-cmp-002-improves-seizure-threshold-and-severity-in-a-model-of-syngap1-related-disorder INAUGURAL SF NIGHT OF IMPACT, CA – 9 days Join us this is our only Gala for 2026! cureSYNGAP1.org/SF26 5TH SCRAMBLE FOR SYNGAP, SC – 137 days Classic case of a small event becoming an institution! cureSYNGAP1.org/Scramble26 CURE SYNGAP1 CONFERENCE - 198 days. cureSYNGAP1.org/Pre26 USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142 PUBMED Pubmed 2026 is at 31. +10 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date SOCIAL MATTERS 4,964 LinkedIn. https://www.linkedin.com/company/curesyngap1 1.58k YouTube. https://www.youtube.com/@CureSYNGAP1 11.1k Twitter https://twitter.com/cureSYNGAP1 45k Insta https://www.instagram.com/curesyngap1 $CAMP closed at $4.56 yesterday. https://www.google.com/finance/beta/quote/CAMP:NASDAQ Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10 Episode 208 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

May 8, 2026Episode 2079 min

Earnings reports: CAMP4 files. Stoke optimizing. Praxis on track. 2026 Video! #S10e207

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 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 which positions us to initiate a global first-in-human Phase 1/2 clinical trial in the second half of 2026. Stoke 1Q26 Results: $411M https://investor.stoketherapeutics.com/news-releases/news-release-details/stoke-therapeutics-announces-first-quarter-2026-financial Lead optimization is underway to identify a clinical candidate for the treatment of SYNGAP1 in 2026. SYNGAP1 is a severe and rare genetic neurodevelopmental disease. Praxis 1Q26 Results: $1.4B https://investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-provides-corporate-update-and-19 Praxis remains on track to nominate a development candidate for each of its three early stage ASO therapeutic initiatives in the first half of 2026: PRAX-090 is designed to address SYNGAP1 loss-of-function (LoF) mutations, a leading cause of severe intellectual disability and epilepsy in DEEs. #CompetitionIsGoodForThePatient KCNT1 Big Day today - Congratulations Post: https://www.linkedin.com/posts/graglia_kcnt1-share-7458560855744618496-X10d Press Release: https://www.linkedin.com/posts/kcnt1-kcnt1-epilepsy-ugcPost-7458487179149787136-kH7c WATCH & SHARE OUR VIDEO Watch and share the new SYNGAP1 Video, it is excellent and helps explain our cause to families and friends. We need to turn them into supporters and donors. cureSYNGAP1.org/SYNGAP1 -> https://youtu.be/pO3ayASBiEk INAUGURAL SF NIGHT OF IMPACT, CA – 20 days Join us this is our only Gala for 2026! cureSYNGAP1.org/SF26 5TH SCRAMBLE FOR SYNGAP, SC – 148 days Classic case of a small event becoming an institution! cureSYNGAP1.org/Scramble26 USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142 PUBMED Pubmed 2026 is at 28. +9 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date https://pubmed.ncbi.nlm.nih.gov/42093631/ ADHD in patients with #DLG2 #NRXN1 #SHANK3 #SYNGAP1 SOCIAL MATTERS 4,940 LinkedIn. https://www.linkedin.com/company/curesyngap1 1,569 YouTube. https://www.youtube.com/@CureSYNGAP1 11.1k Twitter https://twitter.com/cureSYNGAP1 45k Insta https://www.instagram.com/curesyngap1 $CAMP closed at $4.46. https://www.google.com/finance/beta/quote/CAMP:NASDAQ $STOK closed at $32.89. https://www.google.com/finance/beta/quote/STOK:NASDAQ $ACAD closed at $22.40. https://www.google.com/finance/beta/quote/ACAD:NASDAQ $PRAX closed at $330.02. https://www.google.com/finance/beta/quote/PRAX:NASDAQ Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10 Episode 207 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

May 6, 2026Episode 2069 min

Longitudinal Data Matters! Prioritized list of studies! #Sprint4SynGAP a great success! #S10e206

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 https://www.linkedin.com/posts/curesyngap1_curesyngap1-syngap1-rarediseaseresearch-activity-7457507565527044096-dDEK Longitudinal Data Matters - Every six months or more! Study list! CURE SYNGAP1 CONNECT http://curesyngap1.org/connect Citizen Health https://www.citizen.health/ai-advocate/syngap1 Combined Brain ProMMiS https://www.linkedin.com/feed/update/urn:li:activity:7450196488300728320 & Rare-X, the same week. OR DSC. #S10e CURE-ID for Drug responses. Webinar: Thu May 7, 2026 1:30pm – 3pm (PDT) cureSYNGAP1.org/cureID 6th ANNUAL SPRINT FOR SYNGAP1, we raised over $300k! (Gross) Thank you Tavillas for raising over $170k this will go to the Missense fund. We spent over $¼M on missense last year. Thank you Emily Barnes for hosting a New England event for families. https://www.linkedin.com/posts/ecarlisle_we-had-an-incredible-sprint-for-syngap-ugcPost-7457845816502845440-nJCd Thank you to Sara and Sarah for the Virginia event. GREAT WORK. https://www.linkedin.com/posts/sarah-sakly-648544337_syngap1-raredisease-curesyngap1-ugcPost-7456869206341464065-VESz Thank your Rifton for the donation of the tricycle. Congratulations Matthew! https://www.linkedin.com/posts/curesyngap1_syngap1-curesyngap1-sprint4syngap-activity-7457806850676281344--JR- Thank you to the Edouard Family! cureSYNGAP1.org/Crafts26 https://www.linkedin.com/posts/curesyngap1_curesyngap1-syngap1-craftsforacure-activity-7457211260242411520-ekPz INAUGURAL SF NIGHT OF IMPACT, CA – 22 days Join us this is our only Gala for 2026! cureSYNGAP1.org/SF26 5TH SCRAMBLE FOR SYNGAP, SC – 150 days Classic case of a small event becoming an institution! cureSYNGAP1.org/Scramble26 USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142 PUBMED Pubmed 2026 is at 27. +10 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date SOCIAL MATTERS 4,931 LinkedIn. https://www.linkedin.com/company/curesyngap1 1.56k YouTube. https://www.youtube.com/@CureSYNGAP1 11.1k Twitter https://twitter.com/cureSYNGAP1 45k Insta https://www.instagram.com/curesyngap1 $CAMP closed at $3.97 Friday. https://www.google.com/finance/beta/quote/CAMP:NASDAQ Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10 Episode 206 of #Syngap10 #CureSYNGAP1 #Podcast

April 21, 2026Episode 2059 min

Comment on Haystack Petition to FDA. New Board of Trustees. #ICD10 F78.A1 #CureID #S10e205

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: 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: https://www.reuters.com/sustainability/boards-policy-regulation/rare-disease-advocacy-group-urges-trump-administration-restore-fda-clarity-2026-04-01/ Pink Sheet: https://insights.citeline.com/pink-sheet/pathways-and-standards/review-pathways/could-structured-not-ad-hoc-us-fda-flexibility-increase-rare-disease-development-certainty-BYXJENIJLFEINOSO72RS53ZJHE/ Show your support here: https://www.regulations.gov/document/FDA-2026-P-3666-0001 Paragraph 1 – Share information about you and SYNGAP1. Paragraph 2 – Seizures are hard to count while X, Y and Z are major burdens but FDA wants nice countable seizures, this makes it hard to develop drugs. Paragraph 3 – How could “clinically meaningful” endpoints potentially help your community and drug developers? How could a study design other than a ‘randomized clinical trial’ help? How could FDA consulting with disease-specific experts help? Closing – Finish your letter with anything along these lines: We support the framework for all rare set diseases in Haystack’s petition. We don’t believe FDA has to lower the evidentiary bar to approve treatments for our diseases. Randomization isn’t always possible. New scientific methods should be considered. Endpoints specific to our disease should be considered. We urge FDA to open a rulemaking so we can have a legally binding regulation. Board changes, thank you to everyone. https://www.linkedin.com/posts/curesyngap1_curesyngap1-syngap1-patientadvocacy-activity-7450528611339616256-4MJF?utm_source=share&utm_medium=member_desktop&rcm=ACoAAAAD8f4B7JC4TMss45Q8hrsq5kiceI0Z8HE Press Release cureSYNGAP1.org/PR45 US, use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142 Study list! Citizen Health https://www.citizen.health/ai-advocate/syngap1 Combined Brain (May 1 & 2 in NorCal), https://docs.google.com/presentation/d/1IjaHILXj7AlBDlbTJgvYrkBS_0bnI8VCnTIiPXJ7JGM/edit?usp=sharing ProMMiS https://www.linkedin.com/feed/update/urn:li:activity:7450196488300728320 Rare-X, the same week. DSC and Cook’s are coming soon! CURE-ID for Drug responses. CURE-ID is cool. https://cure.ncats.io/home (Webinar coming) Webinar: Thu May 7, 2026 1:30pm – 3pm (PDT) cureSYNGAP1.org/cureID 6th ANNUAL SPRINT FOR SYNGAP1, EVERYWHERE – 5 days - $207k! Go Tavilla. 17 teams raised $265K last year; this year, we have 20+ teams! https://curesyngap1.org/calendar/sprint4syngap-2026 Thank your Rifton for the donation of the tricycle. Email today: https://mailchi.mp/curesyngap1.org/sprint-for-syngap-2026-one-community-one-goal?e=17610baa03 INAUGURAL SF NIGHT OF IMPACT, CA – 38 days Join us this is our only Gala for 2026! cureSYNGAP1.org/SF26 5TH SCRAMBLE FOR SYNGAP, SC – 166 days Classic case of a small event becoming an institution! cureSYNGAP1.org/Scramble26 PUBMED Pubmed 2026 is at 26. +9 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date SOCIAL MATTERS 4,891 LinkedIn. https://www.linkedin.com/company/curesyngap1 1.55k YouTube. https://www.youtube.com/@CureSYNGAP1 11.1k Twitter https://twitter.com/cureSYNGAP1 45k Insta https://www.instagram.com/curesyngap1 $CAMP closed at $4.67 Friday. https://www.google.com/finance/beta/quote/CAMP:NASDAQ Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10 Episode 205 of #Syngap10 #CureSYNGAP1 #Podcast