CURE SYNGAP1 Podcast

Hosted by Mike Graglia, CURE SYNGAP1

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209

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Jun 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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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 $300 was our match. Industry: MultipleAcademics & Clinicians: Stanford, Berkeley & UCSF. Cross-pollination is always good.Speakers: Ash, John, Kathryn, Helen Willsey & Me. Only got a video of John, which was a mistake. If you took one, please share. Here is John: https://www.linkedin.com/posts/graglia_still-reflecting-on-our-inaugural-cure-syngap1-ugcPost-7467439971294048256-cUf9/ Dr. Willsey Rocks.Willsey Press Release https://www.eurekalert.org/news-releases/1130924 (both were at NoI).A few other points on HRW, as we call her. Simons: https://curesyngap1.org/blog/future-research-for-syngap1-how-helen-willsey-broke-new-ground-frogs-in-hand/Willsey in Neuron 2021: https://www.cell.com/neuron/pdf/S0896-6273(21)00002-7.pdf (Frogs)Birtele in Nature Neuroscience 2023: https://www.nature.com/articles/s41593-023-01477-3 (Confirms)McCluskey in Nature Communications 2025: https://www.nature.com/articles/s41467-025-57342-3 (GI)Kostyanovskaya in BioRxiv 2025: https://pubmed.ncbi.nlm.nih.gov/39677731 (cilium)5TH SCRAMBLE FOR SYNGAP, SC – 114 daysClassic case of a small event becoming an institution!cureSYNGAP1.org/Scramble26CURE SYNGAP1 CONFERENCE - 175 dayscureSYNGAP1.org/Pre USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142PUBMEDPubmed 2026 is at 35. +11 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=dateSOCIAL MATTERS5,045 LinkedIn. https://www.linkedin.com/company/curesyngap11.58k YouTube. https://www.youtube.com/@CureSYNGAP111.1k Twitter https://twitter.com/cureSYNGAP145k Insta https://www.instagram.com/curesyngap1$CAMP closed at $4.34 today.https://www.google.com/finance/beta/quote/CAMP:NASDAQLike and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10Episode 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 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 SYNGAP1.1. 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-jfWF2. Send IMPACT REPORT - Tool for family, newly diagnosed & Fundraising.cureSYNGAP1.org/Impact or cureSYNGAP1.org/Impact253. Ask to Donatecuresyngap1.org/donate/CAMP4Joining 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-disorderINAUGURAL SF NIGHT OF IMPACT, CA – 9 daysJoin us this is our only Gala for 2026!cureSYNGAP1.org/SF265TH SCRAMBLE FOR SYNGAP, SC – 137 daysClassic case of a small event becoming an institution!cureSYNGAP1.org/Scramble26CURE SYNGAP1 CONFERENCE - 198 days.cureSYNGAP1.org/Pre26USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142PUBMEDPubmed 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=dateSOCIAL MATTERS4,964 LinkedIn. https://www.linkedin.com/company/curesyngap11.58k YouTube. https://www.youtube.com/@CureSYNGAP111.1k Twitter https://twitter.com/cureSYNGAP145k Insta https://www.instagram.com/curesyngap1$CAMP closed at $4.56 yesterday.https://www.google.com/finance/beta/quote/CAMP:NASDAQLike and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10Episode 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 19CURE SYNGAP1 STRATEGY WORKINGOur 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 REPORTSCAMP4 1Q26 Results: $99M https://investors.camp4tx.com/news-releases/news-release-details/camp4-reports-first-quarter-2026-financial-results-and-corporateWe 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: $1.4B https://investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-provides-corporate-update-and-19 Praxis remains on track to nominate a development candidate for each of its three early stage ASO therapeutic initiatives in the first half of 2026: PRAX-090 is designed to address SYNGAP1 loss-of-function (LoF) mutations, a leading cause of severe intellectual disability and epilepsy in DEEs.#CompetitionIsGoodForThePatientKCNT1 Big Day today - CongratulationsPost: https://www.linkedin.com/posts/graglia_kcnt1-share-7458560855744618496-X10dPress Release: https://www.linkedin.com/posts/kcnt1-kcnt1-epilepsy-ugcPost-7458487179149787136-kH7cWATCH & SHARE OUR VIDEOWatch and share the new SYNGAP1 Video, it is excellent and helps explain our cause to families and friends. We need to turn them into supporters and donors.cureSYNGAP1.org/SYNGAP1 -> https://youtu.be/pO3ayASBiEkINAUGURAL SF NIGHT OF IMPACT, CA – 20 daysJoin us this is our only Gala for 2026!cureSYNGAP1.org/SF265TH SCRAMBLE FOR SYNGAP, SC – 148 daysClassic case of a small event becoming an institution!cureSYNGAP1.org/Scramble26USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142PUBMEDPubmed 2026 is at 28. +9 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=datehttps://pubmed.ncbi.nlm.nih.gov/42093631/ ADHD in patients with #DLG2 #NRXN1 #SHANK3 #SYNGAP1SOCIAL MATTERS4,940 LinkedIn. https://www.linkedin.com/company/curesyngap11,569 YouTube. https://www.youtube.com/@CureSYNGAP111.1k Twitter https://twitter.com/cureSYNGAP145k Insta https://www.instagram.com/curesyngap1$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:NASDAQLike and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10Episode 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 19Congrats to GETA, CURE SYNGAP1 Australia & CAMP4 for a great weekend. https://www.linkedin.com/posts/syngap1-epilepsy-ugcPost-7457791427486466048-j8SBWEBINARS!CURE-ID Webinar tomorrow! Register now. Thu May 7, 2026 1:30pm – 3pm (PDT) cureSYNGAP1.org/cureIDNext 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-dDEKLongitudinal Data Matters - Every six months or more!Study list!CURE SYNGAP1 CONNECT http://curesyngap1.org/connectCitizen Health https://www.citizen.health/ai-advocate/syngap1Combined BrainProMMiS https://www.linkedin.com/feed/update/urn:li:activity:7450196488300728320 & Rare-X, the same week.OR DSC. #S10eCURE-ID for Drug responses. Webinar: Thu May 7, 2026 1:30pm – 3pm (PDT) cureSYNGAP1.org/cureID6th 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 $¼M on missense last year.Thank you Emily Barnes for hosting a New England event for families. https://www.linkedin.com/posts/ecarlisle_we-had-an-incredible-sprint-for-syngap-ugcPost-7457845816502845440-nJCdThank you to Sara and Sarah for the Virginia event. GREAT WORK. https://www.linkedin.com/posts/sarah-sakly-648544337_syngap1-raredisease-curesyngap1-ugcPost-7456869206341464065-VESzThank your Rifton for the donation of the tricycle. Congratulations Matthew! https://www.linkedin.com/posts/curesyngap1_syngap1-curesyngap1-sprint4syngap-activity-7457806850676281344--JR-Thank you to the Edouard Family! cureSYNGAP1.org/Crafts26https://www.linkedin.com/posts/curesyngap1_curesyngap1-syngap1-craftsforacure-activity-7457211260242411520-ekPzINAUGURAL SF NIGHT OF IMPACT, CA – 22 daysJoin us this is our only Gala for 2026!cureSYNGAP1.org/SF265TH SCRAMBLE FOR SYNGAP, SC – 150 daysClassic case of a small event becoming an institution!cureSYNGAP1.org/Scramble26USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142PUBMEDPubmed 2026 is at 27. +10 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=dateSOCIAL MATTERS4,931 LinkedIn. https://www.linkedin.com/company/curesyngap11.56k YouTube. https://www.youtube.com/@CureSYNGAP111.1k Twitter https://twitter.com/cureSYNGAP145k Insta https://www.instagram.com/curesyngap1$CAMP closed at $3.97 Friday.https://www.google.com/finance/beta/quote/CAMP:NASDAQLike and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10Episode 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 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.pdfPetition: 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.pdfReuters 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-3666-0001Paragraph 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=ACoAAAAD8f4B7JC4TMss45Q8hrsq5kiceI0Z8HEPress Release cureSYNGAP1.org/PR45US, use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142Study list!Citizen Health https://www.citizen.health/ai-advocate/syngap1Combined 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 $265K last year; this year, we have 20+ teams!https://curesyngap1.org/calendar/sprint4syngap-2026Thank your Rifton for the donation of the tricycle.Email today: https://mailchi.mp/curesyngap1.org/sprint-for-syngap-2026-one-community-one-goal?e=17610baa03INAUGURAL SF NIGHT OF IMPACT, CA – 38 daysJoin us this is our only Gala for 2026!cureSYNGAP1.org/SF265TH SCRAMBLE FOR SYNGAP, SC – 166 days Classic case of a small event becoming an institution!cureSYNGAP1.org/Scramble26PUBMEDPubmed 2026 is at 26. +9 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=dateSOCIAL MATTERS4,891 LinkedIn. https://www.linkedin.com/company/curesyngap11.55k YouTube. https://www.youtube.com/@CureSYNGAP111.1k Twitter https://twitter.com/cureSYNGAP145k Insta https://www.instagram.com/curesyngap1$CAMP closed at $4.67 Friday.https://www.google.com/finance/beta/quote/CAMP:NASDAQLike and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10Episode 205 of #Syngap10 #CureSYNGAP1 #Podcast

April 14, 2026Episode 2049 min

Global Advocacy. #NL51. Snippets! #SYNGAPcensus = 1,761. UT Arlington & Cook Children’s. #S10e204

Tuesday, April 14, 2026 - Week 16Census is 1,761https://curesyngap1.org/blog/syngap1-census-2026-update-54-q1-total-1761Trip to Texas for UT Arlington and Cook Children'sSystems innovation with Dr. Lal and Dr. PerryMy post: https://www.linkedin.com/posts/graglia_raredisease-activity-7448087944466259970-MADW?utm_source=share*** Dr. Lal’s post: *** https://www.linkedin.com/posts/dennis-lal-71a8988a_epilepsies-neurodevelopmentaldisorders-raredisease-activity-7449111793991577600-sfXZProMMiS patients - 164 unique patients, 96 of whom have had 2 visits & 46 others 3-5 visitshttps://curesyngap1.org/resources/studies/syngap1-prommisCitizen - 289 registeredhttps://www.citizen.health/ai-advocate/syngap1Victoria Arteaga representing CURE SYNGAP1 at IBE Global Leaders Meeting https://www.linkedin.com/posts/victoria-arteaga-26913433_syngap1-rareepilepsy-dee-activity-7449587651885621248-UD9vNewsletter 51 www.cureSYNGAP1.org/NL51SYNGAP1 Snippets You are in this for life.Board meetings to approve budgets, fundraising has never been more important.6th ANNUAL SPRINT FOR SYNGAP1, EVERYWHERE – 12 days - $169k! Go Tavilla.17 teams raised $265K last year; this year, we have 24 teams who have raised almost $170K so far.https://curesyngap1.org/calendar/sprint4syngap-2026INAUGURAL SF NIGHT OF IMPACT, CA – 46 daysJoin us this is our only Gala for 2026!cureSYNGAP1.org/SF265TH SCRAMBLE FOR SYNGAP, SC – 174 days Classic case of a small event becoming an institution!cureSYNGAP1.org/Scramble26PUBMEDPubmed 2026 is at 24. +8 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=dateSOCIAL MATTERS4,869 LinkedIn. https://www.linkedin.com/company/curesyngap11.55k YouTube. https://www.youtube.com/@CureSYNGAP111.1k Twitter https://twitter.com/cureSYNGAP145k Insta https://www.instagram.com/curesyngap1$CAMP closed at $4.64 yesterday.https://www.google.com/finance/beta/quote/CAMP:NASDAQLike and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10Episode 204 of #Syngap10 #CureSYNGAP1 #Podcast

April 4, 2026Episode 2039 min

DSC-III and Data - CURE SYNGAP1 is working tirelessly to increase SYNGAP1 Knowledge #S10e203

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-groupsMore 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 ProMMiS Doctors: Knowles at Stanford / Dr. Abbott at Colorado. AND, excitingly, these two new locations:Rush University Medical Center (Rush) led by Dr. Dr. Liz Berry-Kravis.University of Alabama at Birmingham led by Dr. Martina Bebin.Interestingly we are paired with PMS aka SHANK3 so the comparisons will naturally arise. Both post-synaptic, they are a half step ahead of us. (We will catch up!) SHANK3 and SYNGAP1 have lots in commons: PSD, Synaptic Plasticity, mTOR. Differences, we have more epilepsy, they have more Catatonia (see Table 2 in Trelles 2026 https://pubmed.ncbi.nlm.nih.gov/41895438/). After that I met with Dr. Xin Tang from BCH who is working on some exciting potential therapies and then over to CAMP4, who is moving at light speed.ILAE Rare Epilepsy Big Data Task Force for 4 years! Makes me think about data…Where does SYNGAP1 Data Live?Citizen.Health RetrospectiveProMMiS ClinicalRare-X PRONow the DSC will have bothCombined Brain Registry and EEG Database.In Argentina SYNGAP1 Registry, potentially expanding to Chile and Colombia.In the EU there is PATRE part of EURAS.In the UK, it seems largely via NHS.In China, I don’t know but this paper shows us someone has 99 people: https://pubmed.ncbi.nlm.nih.gov/41914539/Where else?SYNGAP1 is having a moment, we need project manager volunteers.SPRINT FOR SYNGAP1, EVERYWHERE – 21 days - $132k!Get on the map! https://curesyngap1.org/calendar/sprint4syngap-2026INAUGURAL SF NIGHT OF IMPACT, CA – 55 daysJoin us this is our only Gala for 2026!cureSYNGAP1.org/SF265TH SCRAMBLE FOR SYNGAP, SC – 183 days Classic case of a small event becoming an institution!cureSYNGAP1.org/Scramble26PUBMEDPubmed 2026 is at 22. https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=dateSOCIAL MATTERS4,822 LinkedIn. https://www.linkedin.com/company/curesyngap11.55k YouTube. https://www.youtube.com/@CureSYNGAP111.1k Twitter https://twitter.com/cureSYNGAP145k Insta https://www.instagram.com/curesyngap1$CAMP closed at $4.47 today. https://www.google.com/finance/beta/quote/CAMP:NASDAQLike and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10Episode 203 of #Syngap10 #CureSYNGAP1 #Podcast

March 26, 2026Episode 2029 min

It is time to get ready for clinical trials, this is how. #S10e202

Thursday, March 26, 2026 - Week 13Thanks 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 TRIALS1. Stay Connected to CURE SYNGAP1Fill in the Connect Form https://lnkd.in/grjd-H9HHave an annual call with LaurenSubscribe to the CURE SYNGAP1 Podcast everywhere and to our YouTube. 2. Know your mutation, have your genetic report. Memorize it or get a tattoo.3. Sign up for our Natural History StudiesLooking forward: ProMMiS https://lnkd.in/gh7t4s52 (Also helps you figure out travel).Looking back: Citizen Health https://lnkd.in/gKxg33QT4. Participate in Research, get your mutation affirmed and published.Join the CB BioRepository https://lnkd.in/giFhaGmpGive samples early and often, like me last week: https://lnkd.in/gfqKzNZbDo Surveys5. Educate yourself, start with these two blogshttps://curesyngap1.org/blog/emerging-medicines-syngap1-related-disorders-primer-comparison-glossaryhttps://curesyngap1.org/blog/preparing-for-syngap1-clinical-trials-what-families-need-to-know6. Extra Credit: Collect your EEGsGet them from everywhere you have been (check Citizen to be sure).Keep them handy on a google drive… like this: https://lnkd.in/g4DfkKAU Upload them to the CB EEG Repository. Email Lauren for info.#S10e123 https://lnkd.in/gxAFEnip7. Super Extra Credit: If you have a missense, intronic or other weird mutation. We should make a cell line which will allow further study. These cost ~$10k each, so we need to do a fundraiser, but we can help.#S10e SPRINT FOR SYNGAP1, EVERYWHERE – 29 daysGet on the map! https://curesyngap1.org/calendar/sprint4syngap-2026INAUGURAL SF NIGHT OF IMPACT, CA – 63 daysJoin us this is our only Gala for 2026!cureSYNGAP1.org/SF265TH SCRAMBLE FOR SYNGAP, SC – 191 days Classic case of a small event becoming an institution!cureSYNGAP1.org/Scramble26SYNGAP1 AwarenessMust watch this episode of Kelly and Kyle. Careful with those ASMs that challenge bone growth… https://curesyngap1.org/bonesPUBMEDPubmed 2026 is at 18. Some great papers, but will discuss later. https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=dateSOCIAL MATTERS4,786 LinkedIn. https://www.linkedin.com/company/curesyngap11,540 YouTube. https://www.youtube.com/@CureSYNGAP111.1k Twitter https://twitter.com/cureSYNGAP145k Insta https://www.instagram.com/curesyngap1$CAMP stock is at $4.75 on 25 Mar. ‘26 https://www.google.com/finance/beta/quote/CAMP:NASDAQLike and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10Episode 202 of #Syngap10 #CureSYNGAP1 #Podcast

March 6, 2026Episode 2019 min

We need to raise funds for the critical work of CURE SYNGAP1, join us with #Sprint4SYNGAP #S10e201

Friday, March 6, 2026 - Week 10WHAT 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 SYNGAP1Sprint for SYNGAP is coming fast– 49 DAYS. Make a difference. Raise some money.Get on the map! Text sprint26 to 71777https://curesyngap1.org/calendar/sprint4syngap-2026/INAUGURAL SF NIGHT OF IMPACTAlso to raise funds, please join us in SF on May 28th. 83 DAYS. Thanks to the organizational team Justin, Zoe, Ed, Jessica, etc.cureSYNGAP1.org/SF26 NHS MatterI talked in episode 198 #S10e198 about the importance of natural history studies. Check out this paper on Zuvenersen from Dravet to understand the long-term impact these studies could have.https://www.nejm.org/doi/full/10.1056/NEJMoa2506295 Join ProMMiS and Citizen Health.SHOUTOUTSRosie Davilla on Univision curesyngap1.org/rosie2026 >> https://www.univision.com/local/dallas-kuvn/syngap1-el-diagnostico-que-cambio-la-vida-de-rosie-en-texas-video#RareDiseaseDay TalksEmily Barnes @ Quiver; Paulina and Brian Sheehan @ Third Rock; Mike @ SparkNS; John Hill & Allison CNBC Cures.Beata’s double header SYNGAP1 Stories. Part 1.https://curesyngap1.org/podcasts/syngap1-stories/beata-tarasiuk/DSCIIIIn addition to Colorado Children’s & Stanford we are now in a study at Boston Children’s, Rush and U Alabama aka UAB. Attending kick off for this at the end of the month.DATES TO TRACKScramble for Syngap - 5th annual on October 3 in S. Carolina in 211 DAYS cureSYNGAP1.org/Scramble26 Conference in Denver CO! 271 DAYS. Sponsorship options in our #Prospectus for industry are available here https://curesyngap1.org/prospectus Science Day - cureSYNGAP1.org/SD2025VideosFamily Day - cureSYNGAP1.org/FD2025VideosSee our entire library of webinars & videos on YouTube youtube.com/cureSYNGAP1 BIOSAMPLES & EEGs!Biorepository needs more samples. Check out the list and map here https://combinedbrain.org/roadshow/ and contribute both blood & EEGs. The data and research we do with these samples is invaluable. Let us know if you are going, email our CSO@curesyngap1.orgPUBMEDPubmed 2026 is at 12, just like last week but am I seeing some amazing manuscripts! https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date Two particularly cool papers:HDAC Inhibitors https://pubmed.ncbi.nlm.nih.gov/41777621/ A positive missense causing cognitive resiliencehttps://pubmed.ncbi.nlm.nih.gov/41777621/SOCIAL MATTERS4,732 LinkedIn. https://www.linkedin.com/company/curesyngap11,535 YouTube. https://www.youtube.com/@CureSYNGAP111.2k Twitter https://twitter.com/cureSYNGAP145k Insta https://www.instagram.com/curesyngap1$CAMP stock is at $4.59 on 5 Mar. ‘26 https://www.google.com/finance/beta/quote/CAMP:NASDAQLike and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/Episode 201 of #Syngap10 #CureSYNGAP1 #Podcast

February 25, 2026Episode 2009 min

#RareDiseaseWeek! We are doing much beyond grants… too much. Get the Citizen Health App! #S10e200

Wednesday, February 25, 2026 - Week 9Thank 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-IVDQNATURAL HISTORY STUDYSign up for Citizen Health cureSYNGAP1.org/Citizen and ProMMiS cureSYNGAP1.org/ProMMiSAnd now the Citizen Health App on iOS https://www.linkedin.com/posts/graglia_your-advocate-is-now-with-you-in-every-moment-activity-7432260543748579328--dvaBoard meeting… key message, we are much more than fundraising, grants and patient support. Here is our list of non-grant projects.FundraisingRegulatory - IndustryRegulatory - FDAClinical Trial ReadinessStandard of CarePatient EngagementHealth Economics https://www.linkedin.com/posts/graglia_the-economic-impact-of-caregiving-for-individuals-activity-7431827551574011904-HvA4Global CoordinationNext steps with NALLPatient SupportNext steps with NortriptylineBIOSAMPLES & EEGs!Biorepository needs more samples. Check out the list and map here https://combinedbrain.org/roadshow/ and contribute both blood & EEGs. The data and research we do with these samples is invaluable. Let us know if you are going, email our CSO@curesyngap1.orgFUNDRAISING - SPRINT4SYNGAPSprint is April 25 - our calendar page - cureSYNGAP1.org/Sprint - has all the information in the following links:Also, May 28, San Francisco, CA: cureSYNGAP1.org/SF26Scramble for Syngap - 5th annual on October 3 in S. Carolina cureSYNGAP1.org/Scramble26PUBMEDPubmed 2026 is at 9, just like last week but am I seeing some amazing manuscripts! https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date (Remember we had 18 in all of ‘18)SOCIAL MATTERS4,700 LinkedIn. https://www.linkedin.com/company/curesyngap11,530 YouTube. https://www.youtube.com/@CureSYNGAP111.2k Twitter https://twitter.com/cureSYNGAP145k Insta https://www.instagram.com/curesyngap1$CAMP stock is at $4.70 on 24 Feb. ‘26 https://www.google.com/finance/beta/quote/CAMP:NASDAQLike and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/Episode 200 of #Syngap10 #CureSYNGAP1 #Podcast