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

SPRINT FOR SYNGAP1, EVERYWHERE – 29 days

Get on the map!  

https://curesyngap1.org/calendar/sprint4syngap-2026

INAUGURAL SF NIGHT OF IMPACT, CA – 63 days

Join us this is our only Gala for 2026!

cureSYNGAP1.org/SF26

5TH SCRAMBLE FOR SYNGAP, SC – 191 days 

Classic case of a small event becoming an institution!

cureSYNGAP1.org/Scramble26

SYNGAP1 Awareness

Must watch this episode of Kelly and Kyle.  Careful with those ASMs that challenge bone growth… https://curesyngap1.org/bones

PUBMED

Pubmed 2026 is at 18.  Some great papers, but will discuss later. https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date

SOCIAL MATTERS

4,786 LinkedIn.  https://www.linkedin.com/company/curesyngap1

1,540 YouTube.  https://www.youtube.com/@CureSYNGAP1

11.1k Twitter https://twitter.com/cureSYNGAP1

45k Insta https://www.instagram.com/curesyngap1

$CAMP stock is at $4.75 on 25 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 202 of #Syngap10 #CureSYNGAP1 #Podcast

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.

cureSYNGAP1.org/SF26 

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 

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 [email protected]

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, 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.

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 [email protected]

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, 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 [email protected].

(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:

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

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

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

Event: https://www.linkedin.com/posts/curesyngap1_it-was-a-day-to-remembera-perfect-combination-activity-7422379922578587648-wNBP

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, January 16, 2026 - Week 3

Stoke Phase 3 Rapid Recruitment - A timeline that is ours to beat!

If Marcin can make it from Poland, you can make it from where you are!

CAMP4 Website is great and features Dani Williams!

Newsletter is out!

Sara Driscoll Genetic Testing Story - Share it or copy it!

Pubmed 2026 is at 3!

SOCIAL MATTERS

$CAMP stock is at $5.78 on 15 Jan. ‘26

Like and subscribe to this podcast wherever you listen.

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, 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

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, 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

https://www.linkedin.com/posts/shriya-bhat-0b845b203_at-a-patient-advocacy-meeting-in-nashville-activity-7409304451821277184-TO0t 

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