CURE SYNGAP1 PODCAST aka SYNGAP10
SYNGAP10 is a 10 minute weekly blog to keep parents and families up to date on what the team at SynGAP Research Fund (SRF) is doing to advocate for patients& advance research into SYNGAP1. If you do nothing else, listen to or watch this and let us know what you think.
5 days left to register for the conf, #Pipeline, #ProMMiS Protocol, #ORCA Webinar & more #S10e224
Monday, October 26, 2026 — Week 44
DENVER — 5 DAYS TO REGISTER
Science Day 224. Family Day 180, including 33 kids/Syngapians. Family Dinner 57 seats. Registration closes OCT. 31.Register: https://cureSYNGAP1.org/Reg26
Dinner: https://cureSYNGAP1.org/Henrys
Rare Reception: https://cureSYNGAP1.org/RR26 — thank you BioMarin, Lundbeck + Citizen Health.
NEW PIPELINE + COLLABORATION
Updated SYNGAP1 therapeutic pipeline: https://cureSYNGAP1.org/pipeline RAD video — broad collaboration is how rare diseases get treated. https://www.linkedin.com/posts/caleb-moore-4382704_many-terrible-diseases-will-find-treatments-ugcPost-7518359681396989952-8-XH Nordli: Referral + Citizen data + COMBINEDBrain connections. He will speak at the conference.
P...
What You Need to Know & When: DAY-C, UNLOCK, Ari, ORCA, HEOR, Denver (11 days to register) #S10E223
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. [email protected] https://curesyngap1.org/blog/validating-remote-developmental-assessments-syngap1-related-disorders-grant-awarded-julie-orlando-center-for-epilepsy-and-neurodevelopmental-disorders-u-penn-jill-mckee-childrens-hospital-philadelphia/ KNOW YOUR MUTATION. Join Citizen, ask Citizen; get Ari, ask Ari. ~70 families already using Ari. https://cureSYNGAP1.org/Ari COMBIOMICS — if Isaac or Grace from COMBINEDBrain emails you, respond. Very important study.
RAISE FUNDS VIA UNLOCK
$200,000 matching gift! https://curesyngap1.org/calendar/unlock-their-tomorrow-2026-syngap1
COMING SOON — ORCA
ORCA validati...1,871 #SYNGAP1 Census, COMBINEDBrain RR-RGND, Conference Dinner/Drinks & $200K Unlock Match #S10e222
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
Wonderful Rare Research Roundtable on Genetic Neurodevelopmental Disorders. COMBINEDBrain is a gift to the rare neuro community — hug them when you see them. https://combinedbrain.org/RR-RGND Dr. Emma James of Encoded, chair of our IAB, posted about the meeting. https://www.linkedin.com/posts/emma-james-71759a1a_the-combinedbrain-research-roundtable-on-ugcPost-7511223599165472768-jDkI
CURE SYNGAP...
CAMP4 ASCEND Webinar + #SYNGAP1 Goes Global: Argentina, UN, Newborn Screening & Jaxon in ICU #S10E221
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
- CAMP4 + LATAM hybrid meeting: “Path to the Clinical Trial in Argentina.”
- Remember how they got here: THE CONFERENCE. Community → relationships → trial readiness.
- Recording: https://youtu.be/UuhOIzSEKH4
SYNGAP1 LEADERS REPRESENTIN...
ASCEND Trial Webinar + #SYNGAP1 Warriors, Biobank, HEOR & Industry News #S10E220
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, 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: https://cureSYNGAP1.org/ScrambleWARRIORS — KEEP TELLING THE STORIES
NEW: Weston, 22 — diagnosed only 6 months ago. Late diagnosis! https://cureSYNGAP1.org/Warrior Will was Warrior #21 at a...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
SYNGAP...USA Today, Synchrony Conf, #EEC2026, Portugal & Family Resources — #CureSYNGAP1 in Action #S10E218
Tuesday, September 8, 2026 — Week 37
DON’T MISS ASO UPDATEEpisode 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.
https://www.linkedin.com/posts/bernadette-basilico-6924b29a_a-two-year-postdoctoral-position-with-share-7500493855591432192-sOD6/
https://www.usatoday.com/story/life/health-wellness/2026/09/01/kids-special-education-school-shortage-care/90851980007/
SYNCHRONY — PALO ALTO
KAH + I attended...
#Angelman ASO Fails, #AlexanderDisease ASO Wins: #SYNGAP1 CAMP4 ASO? NYT on Clinical Trials #S10E217
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 DISEASEOn 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 driving the disease. Alexander disease is caused by mutations in GFAP. Zilganersen reduces production of abnormal GFAP protein.
http...
Big Week for #SYNGAP1: Trials, Data, #Epilepsy, Palo Alto & Athens #S10E216
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 cash runway through the end of 2028. Q2 earnings / corporate update:https://investors.camp4tx.com/news-releases/news-release-details/camp4-reports-second-quarter-2026-financial-results-and Rare-X + ProMMiS Rare-X is our PRO partner for the ProMMiS Natural History Study. IMPORTANT: Families participating in ProMMiS need to complete their Rare-X surveys within SEVEN DAYS of their ProMMiS Natural History Study visit date...
#AdamsCamp & Tony in Denver: Support, Progress & Open Issues, taking a break from #LakeMary #S10e215
Tony with us for a week after five months away…
Way less screen addicted More independent Chubby - Less exercise, worse food DepressedAdam’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 well, and he was a known quantity to them. The team did a fabulous job. Even though Tony can...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.
Diffe...
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
...
#Future4Ford. 2Q26 #SYNGAP1Census = 1,806 Patients (+45) $CAMP #ValproateDemandBetter #S10e212
#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.
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
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#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 t...#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 we...
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.
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 - Tool for family, newly diagnosed & Fundraising.
cureSYNGAP1.org/Impact or cureSYNGAP1.org/Impact25
Ask to...
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.
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...
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.c...
Global Advocacy. #NL51. Snippets! #SYNGAPcensus = 1,761. UT Arlington & Cook Children’s. #S10e204
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: *** https://www.linkedin.com/posts/dennis-lal-71a8988a_epilepsies-neurodevelopmentaldisorders-raredisease-activity-7449111793991577600-sfXZ
ProMMiS patients - 164 unique patients, 96 of whom have had 2 visits & 46 others 3-5 visits
https://curesyngap1.org/resources/studies/syngap1-prommis
Citize...
DSC-III and Data - CURE SYNGAP1 is working tirelessly to increase SYNGAP1 Knowledge #S10e203
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 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 D...
It is time to get ready for clinical trials, this is how. #S10e202
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 https://curesyngap1.org/connect Have an annual call with Lauren Subscribe to the CURE SYNGAP1 Podcast everywhere and to our YouTube Know your mutation, have your genetic report. Memorize it or get a tattoo. Sign up for our Natural His...We need to raise funds for the critical work of CURE SYNGAP1, join us with #Sprint4SYNGAP #S10e201
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 some money.
Get on the map! Text sprint26 to 71777
https://curesyngap1.org/calendar/sprint4syngap-2026/
INAUGURAL SF NIGHT OF IMPACT
Also to raise funds, please...
#RareDiseaseWeek! We are doing much beyond grants… too much. Get the Citizen Health App! #S10e200
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 ProMMiS cureSYNGAP1.org/ProMMiS
And now the Citizen Health App on iOS https://www.linkedin.com/posts/graglia_your-advocate-is-now-with-you-in-every-moment-activity-7432260543748579328--dva
Board meeting… key message, we are much more than fundraising, grants and patient support. Here is our list of non-grant project...
Support Clinical Trial Design for #SYNGAP1 via biobanks, EEGs and Fundraising. #S10e199
Tuesday, February 17, 2026 - Week 8
We are flat out, thank you to the team who work full-time on SYNGAP1: VM KAH LP PP & KF.
CLINICAL TRIAL DESIGN
We are Angelman-like. (Rett also) https://aesnet.org/abstractslisting/differentiating-key-symptoms-of-angelman-syndrome-as-and-syngap1-via-caregiver-reported-and-us-claims-data-to-understand-differences-between-how-providers-and-caregivers-view-impacts-on-patient-care
Dravet or Angelman? Phase 1/2 is when we try it all. EEGs and NHS help with this effort.
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...
All #SYNGAP1 Families need to take part in our Natural History Studies: ProMMiS & Citizen #S10e198
Thursday, February 5, 2026 - Week 6
Happy #RareDisease & #BlackHistory Month!
#NaturalHistory means how this disease progresses. Reminder: We have only been at this for 17 years, first patients were identified via Hamdan, 2009. https://pubmed.ncbi.nlm.nih.gov/19196676/
Retrospective Digital NHS: cureSYNGAP1.org/Citizen (Growing list of tools available to families, for free)
Prospective Multi-disciplinary Multi-site NHS: ProMMiS cureSYNGAP1.org/ProMMiS
Reminder, only possible by CS1 support for non-CHOP sites and travel plus huge gift to Penn. https://www.chop.edu/news/25-million-gift-penn-medicine-and-children-s-hospital-philadelphia-establishes-center-epilepsy
<...
Funding Priorities. Biorepository. Genetic Testing. How to move for a clinical trail! #S10e197 Monday, February 2, 2026 - Week 6
All US Families, please take the time to fill out the ProMMiS Survey. It takes 5 minutes. It's super important. Even if you haven't been there, we need your feedback.
English: https://curesyngap1.org/SurveyProMMiS
Spanish: https://curesyngap1.org/encuestaProMMiS
Our funding priorities for 2026 are Genetics, Behaviors & Isoforms. https://www.linkedin.com/posts/graglia_syngap1-curesyngap1-grants-activity-7421952845693788160-EHeK
Pubmed 2026 is at 6!
https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date
Coolest paper on Pubmed… GC rich areas were missed...
Stoke Tx shows us that ASO Clinical Development is accelerating. #S10e196
Friday, January 16, 2026 - Week 3
Stoke Phase 3 Rapid Recruitment - A timeline that is ours to beat!
https://investor.stoketherapeutics.com/news-releases/news-release-details/stoke-therapeutics-announces-updates-timelines-completion
If Marcin can make it from Poland, you can make it from where you are!
cureSYNGAP1.org/Marcin
CAMP4 Website is great and features Dani Williams!
https://www.camp4tx.com/
Newsletter is out!
https://cureSYNGAP1.org/NL49
Sara Driscoll Genetic Testing Story - Share it or copy it!
https://cureSYNGAP1.org/Ge...
SYNGAP1 Related Disorder (SRD) Key Talking Points & why we need to use them. #S10e195
Saturday, January 10, 2026 - Week 2
Here are key talking points to reference when talking about SRD. Please let us know your thoughts about how these could be better. https://docs.google.com/document/d/1lXaDQEVwF1K_yAU-RicaJibxb8xoJtldSvnVgopwu00/edit?usp=sharing
SOCIAL MATTERS
4,546 LinkedIn. https://www.linkedin.com/company/curesyngap1/
1,500 YouTube. https://www.youtube.com/@CureSYNGAP1
11.2k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1/
$CAMP stock is at $6.20 on 8 Jan. ‘26 https://www.google.com/finance/beta/quote/CAMP...
Becky Quick launches CNBC Cures, Finding Your Voice & Key Talking Points for SRD! #S10e194
Friday, January 9, 2026 - Week 2
Big news in SYNGAP-land, Becky Quick and Matt Quayle have a beautiful SynGAPian named Kaylie & they are launching CNBC Cures!
- Wonderful to have more awareness of SYNGAP1, I hope it leads to more diagnoses.
- My two favorite quotes from the episode and podcast: “There is no Mission without Money”-BQ & “She has reset our whole life plan…one day I’m going to be gone and is Kaylie going to be ok?”-MQ
- Here is our page with all the links! https://www.linkedin...
Stockdale Paradox: Not getting out at Xmas, but we will win. #SynGAPCensus = 1,707. #S10e193
Friday, January 2, 2026 - Week 1
#SynGAPCensus = 1,707 https://curesyngap1.org/blog/syngap1-census-2025-update-32-q4-2025-1707/
From the Cantor Report on CAMP4
The Stockdale Paradox. The best way to succinctly describe CAMP4 and the parties driving progress in this field (Cure SYNGAP1, families, researchers) is, for anyone familiar with Jim Collins' book "Good to Great," they have fully embraced the "Stockdale Paradox": To succeed in difficult circumstances you must 1) confront the brutal facts (severity of the disorder, devastating
impact on patients and families, lack of treatment) while 2) maintaining unw...
AES ‘25 was incredible, Fundraising, PRV, Behaviors, Posters/Pubmed & Thank you. #S10e192
Saturday, December 20, 2025 - Five days till Christmas, 11 days left to raise funds to CURE SYNGAP1
AES was exceptional in many ways, here are a few:
Rare & SYNGAP1 were both very visible, posters with our Logo and names of staff were seen! Posters: https://www.linkedin.com/posts/graglia_syngap1-curesyngap1-activity-7408291479187755008-rMru Our conference was standing room only and had investors! Even got a mention in their research report! https://www.investing.com/news/analyst-ratings/cantor-fitzgerald-reiterates-overweight-rating-on-camp4-therapeutics-stock-93CH-4403281 ProMMiS Launch was a massive win for patients. Collaboration. Praxis and Lundbeck recruited for exciting drugs and CAMP4 ta...New book by #SYNGAP1 mom for and about #GlassChild (Sibling of Special Needs) #S10e191
#CyberMonday, December 1, 2025. Week 49.
Go buy The Monster Inside My Brother by Nicole Ciccone illustrated by Lena Bardy.
Bookshop: https://bookshop.org/p/books/the-monster-inside-my-brother-nicole-ciccone/8986b9c64d1ebe5d?ean=9798999980700&next=t&
Barnes and Noble: https://www.barnesandnoble.com/w/the-monster-inside-my-brother-nicole-m-ciccone/1148368158
Amazon: https://a.co/d/imfxmwG
See you Thursday at the CURE SYNGAP1 Conference 2025 Atlanta: https://curesyngap1.org/events/conferences/cure-syngap1-conference-2025-hosted-by-srf/
SOCIAL MATTERS
4,474 LinkedIn. https://www.linkedin.com/company/curesyngap1/
1,480 YouTube. https://www.youtube.com/@CureSYN...
AAV. Resources on cureSYNGAP1.org. Congrats to Ultragenyx & Novartis. Conf is in 5 days! #S10e190
Happy Thanksgiving… Thursday, November 27, 2025. Week 48.
Continued from #S10e189…
And the AAV Paper (https://pubmed.ncbi.nlm.nih.gov/40988338/) from #S10e187… https://curesyngap1.org/podcasts/syngap10/clinical-research-ai-dx-nl47-survey-autism-press-6-days-to-register-for-syngap1conf-s10e187/
https://curesyngap1.org/blog/
Issac’s story, Transmitter reprint, Scramble 4 write up and JK on #Autism, #MustRead
https://curesyngap1.org/resources/webinars/ 119 - 112
Register for livestream of the conference, AAV from Allen Inst., dos en espanol, Missense, Unlock and Rare-X for ProMMiS.
https://curesyngap1.o...
#CureSYNGAP1 Conf: 1 week! #GivingTuesday: 5 days. #ProMMiS. Mvmt & Sleep papers. #S10e189
Happy Thanksgiving… Thursday, November 27, 2025. Week 48.
https://curesyngap1.org/clinical-trials/ Emerald from Praxis for Relutrigine and DeeP Ocean from Longboard for Bexicaserin. Check them out, screen for both. See what you can do!
Hectic with #MIHealthSummit, Tony at home, an NHS visit, Ultrabootcamp, not to mention prepping for the most elaborate conference yet!
MI https://www.linkedin.com/posts/raymond-puerini_medtech-patientengagement-mihealthsummit-activity-7392679496266436608-7kWo
Bootcamp https://www.linkedin.com/posts/graglia_rarebootcamp-syngap1-precisionmedicines-activity-7396249525214031872-RvsX
ProMMiS - Meet committed clinicians, get great care and build the future of...
It’s time to UNLOCK THEIR TOMORROW - let’s raise funds. Also: CAMP4 and our name. #S10e188
Sunday, November 9, 2025. Week 46.
End of year campaign: UNLOCK THEIR TOMORROW, curesyngap1.org/unlock
Why now? UNLOCK It’s giving season. Count our blessings and share what we have. CS1 has moved mountains this year: Staff, ProMMiS, etc. But we are just starting…
Every single family should give and should solicit their friends. Yes, it’s a tough time. Yes, people have other causes. No (most of) your friends don’t have a SynGAPian.
Miss 100% of the shots you don’t take. And remember, donors learn about what they give...
Clinical Research, AI Dx, #NL47, Survey, #Autism Press, 6 Days to Register for #SYNGAP1conf #S10e187
Saturday, October 25, 2025. Week 43.
Time to advocate, ELF on the Hill, support available, apply now: https://www.linkedin.com/posts/everylifeorg_were-excited-to-join-everyone-on-capitol-activity-7384625926333943808-mO1U/
PUBMED at 47, and low. Clinical research!
https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2025-2025&sort=date
- CHOP EEG ProMMiS https://www.neurology.org/doi/10.1212/WNL.0000000000214148?url_ver=Z39.88-2003&rfr_id=ori:rid:crossref.org&rfr_dat=cr_pub%20%200pubmed
- COMMUNICATION #ORCA https://acamh.onlinelibrary.wiley.com/doi/full/10.1111/jcpp.70063
Doing surveys...
#SyngapCenus 1,675 Teacher Handout CC/VNS Citizen Paper $CAMP starts GLP Tox CIRM funds S1 #S10e186
Friday, October 3, 2025. Week 40.
#SyngapCenus 1,675 https://curesyngap1.org/blog/syngap1-census-2025-update-39-q3-2025-total-1675/
Rachel J. made an educator handout https://curesyngap1.org/blog/supporting-students-with-syngap1-related-disorders/
Sign up for Citizen Health
https://www.citizen.health/partners/srf
CC/VNS Research w/ Citizen https://www.linkedin.com/posts/citizen-health-inc_syngap1-dee-raredisease-activity-7378823288950575105-YjP3
Rhymes with recent publication from Dr. Perry on Dravet
https://www.tandfonline.com/doi/full/10.1080/14737175.2025.2562118
Citizen AI Advocate avail. for SYNGAP...
F78.A1,the code for #SYNGAP1, turns 4, make sure we use it. #ICD10 for #RareDisease Update. #S10e185
Wednesday, October 1st, 2025. Week 40.
SYNGAP1 Related Disorders secured an ICD-10 code exactly four years ago today, through the advocacy of SRF and the hard work of volunteers like Hans Schlecht. Our code is F78.A1
Blog: https://curesyngap1.org/blog/syngap1-assigned-its-own-icd-10-code-f78-a1-srf/
Check out #S10e8 to learn more: https://www.youtube.com/watch?v=tZ5s5rQawXg
Read the case study: https://everylifefoundation.org/icd-code-roadmap/#toggle-id-13
Hear from other leaders: https://effieparks.com/podcast/episode-224-the-complicated-world-of-icd10-codes-with-ceo-and-co-founder-of-slc6a1-connect-amber-freed
W...
