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Over 1,600 families are caring for a loved one with the rare disease ”SynGAP” resulting from a variant of the SYNGAP1 gene. This weekly podcast is for them. -A quick summary of the latest news in the space. The host is Mike Graglia, co-founder & managing director of CURE SYNGAP1. CURE SYNGAP1 is a 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 https://cureSYNGAP1.org
Episodes
Friday Mar 06, 2026
Friday Mar 06, 2026
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 join us in SF on May 28th. 83 DAYS. Thanks to the organizational team Justin, Zoe, Ed, Jessica, etc.
NHS Matter
I 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.
SHOUTOUTS
Rosie 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 Talks
Emily 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/
DSCIII
In 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 TRACK
Scramble 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/SD2025Videos
Family Day - cureSYNGAP1.org/FD2025Videos
See 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.org
PUBMED
Pubmed 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 MATTERS
4,732 LinkedIn. https://www.linkedin.com/company/curesyngap1
1,535 YouTube. https://www.youtube.com/@CureSYNGAP1
11.2k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1
$CAMP stock is at $4.59 on 5 Mar. ‘26 https://www.google.com/finance/beta/quote/CAMP:NASDAQ
Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/
Episode 201 of #Syngap10 #CureSYNGAP1 #Podcast
Wednesday Feb 25, 2026
Wednesday Feb 25, 2026
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 projects.
- Fundraising
- Regulatory - Industry
- Regulatory - FDA
- Clinical Trial Readiness
- Standard of Care
- Patient Engagement
- Health Economics https://www.linkedin.com/posts/graglia_the-economic-impact-of-caregiving-for-individuals-activity-7431827551574011904-HvA4
- Global Coordination
- Next steps with NALL
- Patient Support
- Next steps with Nortriptyline
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.org
FUNDRAISING - SPRINT4SYNGAP
Sprint 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/SF26
Scramble for Syngap - 5th annual on October 3 in S. Carolina cureSYNGAP1.org/Scramble26
PUBMED
Pubmed 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 MATTERS
4,700 LinkedIn. https://www.linkedin.com/company/curesyngap1
1,530 YouTube. https://www.youtube.com/@CureSYNGAP1
11.2k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1
$CAMP stock is at $4.70 on 24 Feb. ‘26 https://www.google.com/finance/beta/quote/CAMP:NASDAQ
Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/
Episode 200 of #Syngap10 #CureSYNGAP1 #Podcast
Tuesday Feb 17, 2026
Tuesday Feb 17, 2026
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 these samples is invaluable. Let us know if you are going, email our CSO@curesyngap1.org.
(Stay tuned for another exciting device study…)
NATURAL HISTORY STUDY
Sign up for Citizen Health cureSYNGAP1.org/Citizen and ProMMiS cureSYNGAP1.org/ProMMiS
NHS Survey in English: https://curesyngap1.org/SurveyProMMiS & Spanish: https://curesyngap1.org/encuestaProMMiS
Latest Pod on NHS: https://youtu.be/7W38uWKBIAw?si=lCrffwMXidmYWz7t
FUNDRAISING - SPRINT4SYNGAP
Sprint is April 25 - our calendar page - cureSYNGAP1.org/Sprint - has all the information in the following links:
set up your team - cureSYNGAP1.org/Sprint26
resource guide for your event - cureSYNGAP1.org/S4SGuide
webinar #99 to help get you started - cureSYNGAP1.org/S4S25
Also, May 28, San Francisco, CA: cureSYNGAP1.org/SF26
Scramble for Syngap - 5th annual on October 3 in S. Carolina cureSYNGAP1.org/Scramble26
PUBMED
Pubmed 2026 is at 9! https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date (Remember we had 18 in all of ‘18)
Cool connection to #PraderWilli Syndrome. https://www.linkedin.com/posts/graglia_syngap1-praderwilli-autism-share-7429579885985296385-zuIH
ETC
- More warriors cureSYNGAP1.org/Warrior
- Dr. Donlin-Asp Press Release cureSYNGAP1.org/PR42 see talk here https://www.youtube.com/watch?v=lR8qcZK-9ro
- Bravo Sara Driscol and GeneDx https://www.linkedin.com/posts/genedx_beyondawareforrare-ugcPost-7427763511235248129-QPPL?utm_source=share&utm_medium=member_desktop&rcm=ACoAAAAD8f4B7JC4TMss45Q8hrsq5kiceI0Z8HE
SOCIAL MATTERS
4,686 LinkedIn. https://www.linkedin.com/company/curesyngap1
1,520 YouTube. https://www.youtube.com/@CureSYNGAP1
11.2k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1
$CAMP stock is at $3.85 on 17 Feb. ‘26 https://www.google.com/finance/beta/quote/CAMP:NASDAQ
Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/
Episode 199 of #Syngap10 #CureSYNGAP1 #Podcast
Friday Feb 06, 2026
Friday Feb 06, 2026
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
Potential for being a control arm in the future.
Protocol: https://www.linkedin.com/posts/curesyngap1_syngap1-stxbp1-dee-activity-7425223573134327808-SVEQ & early data: https://pubmed.ncbi.nlm.nih.gov/40119723/
Join the ~160 families who have enjoyed excellent clinical care and contributed tot he future of SYNGAP1. Today, a 4 month old is going!
CHOP: 119 new, V2- 67, V3- 32, V4- 10, V5- 4
CHCO: 37 new, V2- 7
Stanford: 8 new, V2- 2
Total: 164 (double counting one family who goes to multiple sites)
Survey
English: https://curesyngap1.org/SurveyProMMiS Spanish: https://curesyngap1.org/encuestaProMMiS
94 Responses to survey, so far:
Why not? Did not receive an invitation, Too far to travel, Too expensive
Barriers: Logistics, Cost, Time off, Behaviors, Insurance
ETC.
Pubmed 2026 is at 6! But will soon be 7 with the McKee paper!
https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date
Biorepository needs more samples. Check out the list and map here https://docs.google.com/presentation/d/1IjaHILXj7AlBDlbTJgvYrkBS_0bnI8VCnTIiPXJ7JGM/edit?usp=sharing and contribute blood. The data and research we do with these samples is invaluable.
May 28, San Francisco, CA: cureSYNGAP1.org/SF26
SOCIAL MATTERS
4,668 LinkedIn. https://www.linkedin.com/company/curesyngap1/
1,520 YouTube. https://www.youtube.com/@CureSYNGAP1
11.2k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1/
$CAMP stock is at $3.59 on 5 Feb. ‘26 https://www.google.com/finance/beta/quote/CAMP:NASDAQ
Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/
Episode 198 of #Syngap10 #CureSYNGAP1 #Podcast
Monday Feb 02, 2026
Monday Feb 02, 2026
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, so not only do we need to push for testing, but also REtesting.
https://pubmed.ncbi.nlm.nih.gov/41577710/
Bravo to CURE SYNGAP1 Poland (please send me a shirt) & thank you to CAMP4
Don’t move to or from US/EU/LatAm etc. This question keeps coming. Please stay where you are and advocate like crazy. Set up CURE SYNGAP1 [Your location]
Biorepository needs more samples. Check out the list and map here https://docs.google.com/presentation/d/1IjaHILXj7AlBDlbTJgvYrkBS_0bnI8VCnTIiPXJ7JGM/edit?usp=sharing and contribute blood. The data and research we do with these samples is invaluable.
Annual update to Champions of Hope! https://curesyngap1.org/champions-of-hope/
May 28, San Francisco, CA: cureSYNGAP1.org/SF26
SOCIAL MATTERS
4,661 LinkedIn. https://www.linkedin.com/company/curesyngap1/
1,520 YouTube. https://www.youtube.com/@CureSYNGAP1
11.2k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1/
$CAMP stock is at $3.83 on 27 Jan. ‘26 https://www.google.com/finance/beta/quote/CAMP:NASDAQ
Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/
Episode 197 of #Syngap10 #CureSYNGAP1 #Podcast
Friday Jan 16, 2026
Friday Jan 16, 2026
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/GenTest
Pubmed 2026 is at 3!
https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date
SOCIAL MATTERS
4,604 LinkedIn. https://www.linkedin.com/company/curesyngap1/
1,510 YouTube. https://www.youtube.com/@CureSYNGAP1
11.2k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1/
$CAMP stock is at $5.78 on 15 Jan. ‘26
https://www.google.com/finance/beta/quote/CAMP:NASDAQ
Like and subscribe to this podcast wherever you listen.
https://curesyngap1.org/podcasts/syngap10/
Episode 196 of #Syngap10 #CureSYNGAP1 #Podcast
Sunday Jan 11, 2026
Sunday Jan 11, 2026
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:NASDAQ
Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/
Episode 195 of #Syngap10 #CureSYNGAP1 #Podcast
Friday Jan 09, 2026
Friday Jan 09, 2026
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.com/posts/curesyngap1_syngap1-curesyngap1-cnbccures-activity-7415094066675216387-32wF
curesyngap1.org/kaylie
It's important for us all to remember that it can take time to find our voice. And then use it.
Speaking of using our voice, what can you say? We worked yesterday on Key Talking points which will live here and I will talk about them in the next episode. https://docs.google.com/document/d/1lXaDQEVwF1K_yAU-RicaJibxb8xoJtldSvnVgopwu00/edit?usp=sharing
First paper of 2026, Challenges of Caregiving in SYNGAP1, STXBP1, and TSC.
https://pubmed.ncbi.nlm.nih.gov/41405416/
PUBMED is at 1 for the year. https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.1998-2026&timeline=expanded&sort=date
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:NASDAQ
Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/
Episode 194 of #Syngap10 #CureSYNGAP1 #Podcast
Friday Jan 02, 2026
Friday Jan 02, 2026
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 unwavering faith that you can and will prevail in the end. It gives us conviction that there WILL be a therapy approved for SYNGAP sooner than later and CAMP is most likely to deliver it.
Read more on Jim Collins site:
https://www.jimcollins.com/concepts/Stockdale-Concept.html
This is exactly what SYNGAP1 Argentina achieved at our conference. Acting with certainty that they can and will prevail.
Check out their exceptional flyer: https://drive.google.com/file/d/1O_DldABKTkB9ZLIiUBqXGBMrtlzie-7i/view?usp=share_link
PUBMED is at 59 for the year, that is +4 over our best year, last year. 177 since 2022, almost half of our SYNGAP1 Knowledge (366) has been created in the past 4 years!
https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.1998-2026&timeline=expanded&sort=date
#20Posters Speaking of publications, I talked about 16 posters at AES this year and shared on LI, but I was wrong in the responses I realized we are up to 20! https://www.linkedin.com/posts/graglia_syngap1-curesyngap1-activity-7408291479187755008-rMru
Mutation Tattoo Story
SOCIAL MATTERS
4,529 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 $6.00 on 2 Jan. ‘26 https://www.google.com/finance/beta/quote/CAMP:NASDAQ
Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/
Episode 193 of #Syngap10 #CureSYNGAP1 #Podcast
Saturday Dec 20, 2025
Saturday Dec 20, 2025
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 talked about their ASO and recruiting next year.
- Our community's presence was felt well into AES.
Aaron's post on growth! https://www.facebook.com/aaron.j.harding.5/posts/pfbid0231DtMVUtkZa4eXLv8C8qbf4xEN95aRP1xJ8sGNNvun7aDuUyZVatMWUjjigdXfg1l
Pre-register now for Denver: cureSYNGAP1.org/Pre26
Fundraising. We are YTD $1.68M which is below $1.86M in ’23 and $1.97M in ’24. We need to really double down on fundraising for the next two weeks and into next year. Support our campaign at curesyngap1.org/unlock
ACTION ALERT 🚨Call Bernie and urge him to help children at NO COST to the USG by supporting the PRV. (202) 224-5141
PUBMED is at 58 for the year, that is +4 over our best year, last year. 187 since 2022, more than half of our SYNGAP1 Knowledge (365) has been created in the past 4 years!
https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.1998-2026&timeline=expanded&sort=date
SOCIAL MATTERS
4,519 LinkedIn. https://www.linkedin.com/company/curesyngap1/
1,490 YouTube. https://www.youtube.com/@CureSYNGAP1
11.2k Twitter https://twitter.com/cureSYNGAP1
45k Insta https://www.instagram.com/curesyngap1/
$CAMP stock is at $6.38 on 19 Dec. ‘25 https://www.google.com/finance/beta/quote/CAMP:NASDAQ
Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10/
Episode 192 of #Syngap10 #CureSYNGAP1 #Podcast