CURE SYNGAP1 PODCAST aka SYNGAP10

Monday, August 10, 2026 - Week 33

 

Make sure to watch #S10e213 first! https://www.linkedin.com/posts/curesyngap1_camp4-trial-approved-in-australia-argentina-activity-7491598254027984896-wWTg

145 done!  Out of 516 in US per last census: (https://www.linkedin.com/posts/curesyngap1_syngap1census-syngap1-census-activity-7478070129340215296-6Hjx)

 

CLINICAL TRIAL READINESS SURVEY - http://curesyngap1.org/CTRpoll

Do it now, let us figure out the state of our community.

 

CITIZEN Sign up now! https://www.citizen.health/ai-advocate/syngap1

Everyone with a sick kid needs this.

 

Different from CONNECT: https://curesyngap1.org/curesyngap1connect

Make sure we know how to reach you.

STUDIES ARE HAPPENING

- Albus, thank you to those families. Plug it in.
- Combi[o]mics - Thank you for doing this if invited.
- Everyone will get an invite to do an ORCA Validation Study, please do this.  If you don’t get that email by end of August, bug us.  We are only inviting those we can confirm have a mutation.

 

PERSONAL FUNDRAISERS
Fundraisers advance our mission & help you connect to community

#ForAda curesyngap1.org/forada is over $104k of $10k

#4Ford curesyngap1.org/4ford is over $20k of $20k

 

SIBLINGS - Go Porter!
cureSYNGAP1.org/Sibling 

 

THINGS TO LOOK FORWARD TO…

5TH SCRAMBLE FOR SYNGAP, SC – 54 days till October 3rd

Classic case of a small event becoming an institution! cureSYNGAP1.org/Scramble26

 

SHOOT FOR SYNGAP, UT – 96 days till November 14th

curesyngap1.org/calendar/shoot-for-syngap1

 

FIGHT FOR FELIPE, MA - 111 days till November 29th

curesyngap1.org/Fight26

 

CURE SYNGAP1 CONFERENCE - 115 days until December 3rd & 4th

cureSYNGAP1.org/Reg26 rooms available: ‭cureSYNGAP1.org/denhyatt for $159.  In on Wed, out on Saturday.

 

PUBMED

Pubmed 2026 is at 47. +14 vs the week. (61 last year was +9) We are already at the 3rd highest year. https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date

 

USA 🇺🇲: Use your ICD-10, F78.A1

 

SOCIAL MATTERS

5,159 LinkedIn.  https://www.linkedin.com/company/curesyngap1

1.6k YouTube.  https://www.youtube.com/@CureSYNGAP1

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

43.7k Insta https://www.instagram.com/curesyngap1

 

Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10

Episode 214 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

Friday, August 7, 2026 - Week 32

 

CAMP4 - Gaining momentum

- Approval for trials in Australia & +$50M of cash from investors:https://investors.camp4tx.com/news-releases/news-release-details/camp4-therapeutics-secures-australian-regulatory-clearance

- Profile on CNBC Cures: https://www.cnbc.com/2026/08/06/camp4-advances-rare-disease-treatment-for-syngap1-into-human-trials.html Where they mentioned approval in Argentina!

 

$CAMP closed at $4.53 yesterday. https://www.google.com/finance/beta/quote/CAMP:NASDAQ

 

MAKE SURE YOU ARE REGISTERED WITH CONNECT

https://curesyngap1.org/curesyngap1connect/

 

USA 🇺🇲: Use your ICD-10, F78.A1

 

CITIZEN

Sign up now! https://www.citizen.health/ai-advocate/syngap1

 

SOCIAL MATTERS

5,156 LinkedIn.  https://www.linkedin.com/company/curesyngap1

1.6k YouTube.  https://www.youtube.com/@CureSYNGAP1

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

43.7k Insta https://www.instagram.com/curesyngap1

 

Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10

Episode 213 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

#Future4Ford. 2Q26 #SYNGAP1Census = 1,806 Patients (+45) $CAMP #ValproateDemandBetter #S10e212

Saturday, July 4, 2026 - Week 27

 

#Future4Ford! Already at $3,500+!  Share.  Thank you to the Family.

This is the model for newly diagnosed, call us, set up a page, help us help your loved one. cureSYNGAP1.org/4Ford 

 

CENSUS - 1,806 https://curesyngap1.org/blog/syngap1-census-2026-update-45-q2-total-1806/

Per https://docs.google.com/spreadsheets/d/1oJwMysR2wyTxe91zLlKJglNa0NySPxkBF0PRiV6mBmM/edit?usp=sharing, no change in Germany, UK, or Netherlands.  This is clearly wrong.

 

MERCH - 9 days left!

New t-shirts!  Buy for everyone! There are some funny ones this year and the classics. cureSYNGAP1.org/Bonfire

 

CAMP4 has been busy!

Meeting with one of our families: https://www.linkedin.com/posts/syngap1-share-7473374060614201345-SeKO/

Shoutout on NASDAQ: https://www.linkedin.com/posts/camp4-therapeutics_syngap1awareness-regrna-syngap1-activity-7474823632972820480-UMqi 

$CAMP closed at $4.43 yesterday. https://www.google.com/finance/beta/quote/CAMP:NASDAQ

 

BONES – This is not medical advice, I am not a doctor.  Talk to a doctor AND know your facts.

https://curesyngap1.org/blog/navigating-a-lifetime-of-diagnoses-michaels-syngap1-journey-and-the-effects-of-anti-seizure-medications-on-bone-density/

 

Valproate is the active Ion, it comes from Valproic Acid (Depakene), Sodium Valproate or  Divalproex Sodium (Depakote).  They all cause bone loss by stoping stomach from absorbing, then robbing bones, then flushing out.  More and longer is worse.  Vitamin D and Calcium can help but they need to start early and it’s better to just find another drug.  Also insurance likes it because it’s cheap and old – demand better.

 

“Valproic acid and clobazam were commonly used for epilepsy treatment, while risperidone, aripiprazole, and guanfacine were commonly used for behavior management. Valproate and lamotrigine were more effective at reducing seizure frequencies or maintaining seizure freedom than other anti-seizure medications.”  Clinical signatures of SYNGAP1-related disorders through data integration https://pmc.ncbi.nlm.nih.gov/articles/PMC12419475/

 

THINGS TO LOOK FORWARD TO…

5TH SCRAMBLE FOR SYNGAP, SC – 91 days till October 3rd

Classic case of a small event becoming an institution! cureSYNGAP1.org/Scramble26

 

SHOOT FOR SYNGAP, UT – 133 days till November 14th

curesyngap1.org/calendar/shoot-for-syngap1/ 

 

FIGHT FOR FELIPE, MA - 148 days till November 29th

curesyngap1.org/Fight26 

 

CURE SYNGAP1 CONFERENCE - 152 days until December 3rd & 4th

cureSYNGAP1.org/Denver rooms available: ‭cureSYNGAP1.org/denhyatt for $159.  In on Wed, out on Saturday.

 

PUBMED

Pubmed 2026 is at 40. +13 vs the week. (61 last year was +9) We are already at the 4th highest year. https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date

 

Thanks to Dr. Stephen Smith for this exciting paper, especially in the context of ASOs…
Genetic rescue of disrupted synaptic protein interaction network dynamics following SYNGAP1 reactivation https://pubmed.ncbi.nlm.nih.gov/42362191/  

 

USA 🇺🇲: Use your ICD-10, F78.A1

 

SOCIAL MATTERS

5,106 LinkedIn.  https://www.linkedin.com/company/curesyngap1

1.59k YouTube.  https://www.youtube.com/@CureSYNGAP1

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

43.7k Insta https://www.instagram.com/curesyngap1

 

Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10

Episode 212 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

Friday, June 26, 2026 - Week 26

 

BIO was good.  Need a mouse lab to test molecules all year long.  

https://www.linkedin.com/posts/graglia_syngap-syngap1-mousemodels-ugcPost-7476041484408901633-Zldd/ 

 

MDBR was big, not just Justin A.!  Thank you Dr. McKee, Heather and others. cureSYNGAP1.org/MDBR26Recap 

 

MERCH - 19 days left!

New t-shirts!  Buy for everyone! There are some funny ones this year and the classics. cureSYNGAP1.org/Bonfire

 

GENETIC TESTING

Valuable resource as you have questions. curesyngap1.org/GT  

 

WARRIORS - Phoenix and Kenna

cureSYNGAP1.org/Warrior 

 

THINGS TO LOOK FORWARD TO…

 

WEBINAR: Understanding ASOs & Informed Consent

June 30 4:30 ET curesyngap1.org/calendar/understanding-asos-informed-consent/ 

 

5TH SCRAMBLE FOR SYNGAP, SC – 99 days till October 3rd

Classic case of a small event becoming an institution! cureSYNGAP1.org/Scramble26

 

SHOOT FOR SYNGAP, UT – 141 days till November 14th

fb.watch/HRnwf9FnEB/

 

FIGHT FOR FELIPE, MA - 155 days till November 29th

curesyngap1.org/Fight26 

 

CURE SYNGAP1 CONFERENCE - 159 days until December 3rd & 4th

cureSYNGAP1.org/Denver rooms available: ‭cureSYNGAP1.org/denhyatt for $159.  In on Wed, out on Saturday.

 

PUBMED

Pubmed 2026 is at 38. +12 vs the week. (61 last year was +9) We are already at the 4th highest year. https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date

 

Props to Dr. Frazier for yet another paper on SYNGAP1 based on his study, one of the best grants ever.

https://onlinelibrary.wiley.com/doi/10.1111/dmcn.70337

 

USA 🇺🇲: Use your ICD-10, F78.A1

 

SOCIAL MATTERS

5,074 LinkedIn.  https://www.linkedin.com/company/curesyngap1

1.59k YouTube.  https://www.youtube.com/@CureSYNGAP1

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

43.8k Insta https://www.instagram.com/curesyngap1

 

$CAMP closed at $3.92 yesterday.

https://www.google.com/finance/beta/quote/CAMP:NASDAQ

 

Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10

Episode 211 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

Friday, June 19, 2026 - Week 25

 

June 21 is SYNGAP1 Awareness Day, but we have made it SYNGAP1 Awareness month!  Why 6/21? Because 6p21.32  Donate!  cureSYNGAP1.org/Donate 

 

#ThisIsOursToLose & #BurdenHopeProgress watch #S10e208 https://curesyngap1.org/podcasts/syngap10/tony-update-thisisourstolose-more-great-results-from-camp4-nightofimpact-in-9-days-s10e208/ 

 

New family email, very good, lots of questions.  It’s all here.  Reach out, we are here for you and you are welcome in this community – you are part of it whether you like it or not.

• Raise money.  Join our host committee for 2nd SF event next year.
• Believe that we are doing the best we possibly can, and you would be adrift without a PAG.  If you think this is painful, imagine if we weren't here!
• Register for the Conference.  Book tickets to DEN now.  cureSYNGAP1.org/Denver
• Join us -- make this org better for all of us and all our kids for all their lives.

 

Complex work doesn't fit neatly in 10 minutes anymore... Health Economics and Market Access work is going well, we will put out an RFP shortly.

 

NATURAL HISTORY
Goes On!  Make sure you are in it. https://curesyngap1.org/resources/studies/syngap1-prommis/

Orlando/McKee Grant "Validating Remote Developmental Assessments in SYNGAP1-Related Disorders" cureSYNGAP1.org/PR49  

 

🤖 ARI Sign up now, s/he keeps getting better, and then connect to Citizen Health, if you are not on Citizen Health, fix that.

1. https://www.citizen.health/ai-advocate/syngap1
2. https://ari.citizenhealth.com/

 

KUDOS

- The fifth MDBR, Justin Albrecht rode 70 remote in NC.  Raised $2k.

- Rachel Jasiczek (Yah-she-check) was a guest on Remarkable Futures Podcast - https://open.spotify.com/episode/7dYmHcXEu58H4C8VCoZ9V1?si=x3xvui86TY2cyGHyS3mGDQ

 - Collective: CURE SYNGAP1 Collective - cureSYNGAP1.org/PR48  Collective page - cureSYNGAP1.org/Collective 

 

More fundraisers! 
WARD - Shoot for SYNGAP on November 14th in Utah! https://fb.watch/HRnwf9FnEB/

 Another in Boston in the works…

WEBINAR: Understanding ASOs & Informed Consent

June 30 4:30 ET https://curesyngap1.org/calendar/understanding-asos-informed-consent/

 

5TH SCRAMBLE FOR SYNGAP, SC – 106 days till October 3rd

Classic case of a small event becoming an institution!

cureSYNGAP1.org/Scramble26

 

CURE SYNGAP1 CONFERENCE - 166 days until December 3rd

cureSYNGAP1.org/Denver rooms available: ‭cureSYNGAP1.org/denhyatt for $159.  In on Wed, out on Saturday.

 

USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142

 

PUBMED

Pubmed 2026 is at 37. +12 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date

 

SOCIAL MATTERS

5,058 LinkedIn.  https://www.linkedin.com/company/curesyngap1

1.58k YouTube.  https://www.youtube.com/@CureSYNGAP1

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

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

 

$CAMP closed at $4.13 yesterday.

https://www.google.com/finance/beta/quote/CAMP:NASDAQ

 

Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10

Episode 210 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

Wednesday, June 10, 2026 - Week 24

 

#NightOfImpact was 15 Days ago!

Photos: https://jeaniehorton.pixieset.com/curesyngap1nightofimpact2026/

Impact: $800k+, of which $300 was our match. 

Industry: Multiple

Academics & Clinicians: Stanford, Berkeley & UCSF.  Cross-pollination is always good.

Speakers: Ash, John, Kathryn, Helen Willsey & Me.  Only got a video of John, which was a mistake. If you took one, please share.  Here is John: https://www.linkedin.com/posts/graglia_still-reflecting-on-our-inaugural-cure-syngap1-ugcPost-7467439971294048256-cUf9/ 

 

Dr. Willsey Rocks.

Willsey Press Release https://www.eurekalert.org/news-releases/1130924 (both were at NoI).

A few other points on HRW, as we call her. 
Simons: https://curesyngap1.org/blog/future-research-for-syngap1-how-helen-willsey-broke-new-ground-frogs-in-hand/

Willsey in Neuron 2021: https://www.cell.com/neuron/pdf/S0896-6273(21)00002-7.pdf (Frogs)

Birtele in Nature Neuroscience 2023: https://www.nature.com/articles/s41593-023-01477-3 (Confirms)

McCluskey in Nature Communications 2025: https://www.nature.com/articles/s41467-025-57342-3 (GI)
Kostyanovskaya in BioRxiv 2025: https://pubmed.ncbi.nlm.nih.gov/39677731 (cilium)

 

5TH SCRAMBLE FOR SYNGAP, SC – 114 days

Classic case of a small event becoming an institution!

cureSYNGAP1.org/Scramble26

 

CURE SYNGAP1 CONFERENCE - 175 days

cureSYNGAP1.org/Pre 

 

USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142

 

PUBMED

Pubmed 2026 is at 35. +11 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date

 

SOCIAL MATTERS

5,045 LinkedIn.  https://www.linkedin.com/company/curesyngap1

1.58k YouTube.  https://www.youtube.com/@CureSYNGAP1

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

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

 

$CAMP closed at $4.34 today.

https://www.google.com/finance/beta/quote/CAMP:NASDAQ

 

Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10

Episode 209 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

Tuesday, May 19, 2026 - Week 21

 

Personal Update via Syngap Stories:

Page: cureSYNGAP1.org/stories41 

Post: https://www.linkedin.com/posts/syngap1stories-syngap1-curesyngap1-share-7462125026276777984-OVBr

 

#ThisIsOursToLose - Three steps to engaging friends with CURE SYNGAP1.

 

1. Share CURE SYNGAP1 Video - Burden, Hope & Progress - 1.1k views in 11 days.

cureSYNGAP1.org/Burden https://www.linkedin.com/posts/syngap1-curesyngap1-raredisease-share-7458298251893448706-jfWF

 

2. Send IMPACT REPORT - Tool for family, newly diagnosed & Fundraising.

cureSYNGAP1.org/Impact or cureSYNGAP1.org/Impact25

 

3. Ask to Donate

curesyngap1.org/donate/

 

CAMP4

Joining us at the Night of Impact (with Stoke, Acadia, BioMarin, Gondola & others) and…

https://www.biospace.com/press-releases/camp4-therapeutics-to-present-new-preclinical-data-demonstrating-cmp-002-improves-seizure-threshold-and-severity-in-a-model-of-syngap1-related-disorder

 

INAUGURAL SF NIGHT OF IMPACT, CA – 9 days

Join us this is our only Gala for 2026!

cureSYNGAP1.org/SF26

 

5TH SCRAMBLE FOR SYNGAP, SC – 137 days

Classic case of a small event becoming an institution!

cureSYNGAP1.org/Scramble26

 

CURE SYNGAP1 CONFERENCE - 198 days.

cureSYNGAP1.org/Pre26

 

USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142

 

PUBMED

Pubmed 2026 is at 31. +10 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date

 

SOCIAL MATTERS

4,964 LinkedIn.  https://www.linkedin.com/company/curesyngap1

1.58k YouTube.  https://www.youtube.com/@CureSYNGAP1

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

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

 

$CAMP closed at $4.56 yesterday.

https://www.google.com/finance/beta/quote/CAMP:NASDAQ

 

Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10

Episode 208 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

Friday, May 8, 2026 - Week 19

 

CURE SYNGAP1 STRATEGY WORKING

Our strategy has always been to derisk SYNGAP1 so that industry will invest and bring their skill and capital to bear.  We leverage donor dollars to maximize impact.  Yesterday was a great example:

 

EARNINGS REPORTS

CAMP4 1Q26 Results: $99M https://investors.camp4tx.com/news-releases/news-release-details/camp4-reports-first-quarter-2026-financial-results-and-corporate

We submitted our first regulatory filing for CMP-002 in Australia which positions us to initiate a global first-in-human Phase 1/2 clinical trial in the second half of 2026.

 

Stoke 1Q26 Results: $411M https://investor.stoketherapeutics.com/news-releases/news-release-details/stoke-therapeutics-announces-first-quarter-2026-financial 

Lead optimization is underway to identify a clinical candidate for the treatment of SYNGAP1 in 2026. SYNGAP1 is a severe and rare genetic neurodevelopmental disease.

 

Praxis 1Q26 Results: $1.4B https://investors.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-provides-corporate-update-and-19 

Praxis remains on track to nominate a development candidate for each of its three early stage ASO therapeutic initiatives in the first half of 2026:  PRAX-090 is designed to address SYNGAP1 loss-of-function (LoF) mutations, a leading cause of severe intellectual disability and epilepsy in DEEs.

 

#CompetitionIsGoodForThePatient 

 

KCNT1 Big Day today - Congratulations

Post: https://www.linkedin.com/posts/graglia_kcnt1-share-7458560855744618496-X10d

Press Release: https://www.linkedin.com/posts/kcnt1-kcnt1-epilepsy-ugcPost-7458487179149787136-kH7c/

 

WATCH & SHARE OUR VIDEO

Watch and share the new SYNGAP1 Video, it is excellent and helps explain our cause to families and friends.  We need to turn them into supporters and donors.

cureSYNGAP1.org/SYNGAP1 -> https://youtu.be/pO3ayASBiEk

 

INAUGURAL SF NIGHT OF IMPACT, CA – 20 days

Join us this is our only Gala for 2026!

cureSYNGAP1.org/SF26

 

5TH SCRAMBLE FOR SYNGAP, SC – 148 days

Classic case of a small event becoming an institution!

cureSYNGAP1.org/Scramble26

 

USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142

 

PUBMED

Pubmed 2026 is at 28. +9 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date

 

https://pubmed.ncbi.nlm.nih.gov/42093631/ ADHD in patients with #DLG2 #NRXN1 #SHANK3 #SYNGAP1

 

SOCIAL MATTERS

4,940 LinkedIn.  https://www.linkedin.com/company/curesyngap1

1,569 YouTube.  https://www.youtube.com/@CureSYNGAP1

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

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

 

$CAMP closed at $4.46. https://www.google.com/finance/beta/quote/CAMP:NASDAQ

$STOK closed at $32.89. https://www.google.com/finance/beta/quote/STOK:NASDAQ

$ACAD closed at $22.40. https://www.google.com/finance/beta/quote/ACAD:NASDAQ

$PRAX closed at $330.02. https://www.google.com/finance/beta/quote/PRAX:NASDAQ

 

Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10

Episode 207 of #Syngap10 #SYNGAP1 #CureSYNGAP1 #Podcast #PatientAdvocacy

Wednesday, May 6, 2026 - Week 19

 

Congrats to GETA, CURE SYNGAP1 Australia & CAMP4 for a great weekend. https://www.linkedin.com/posts/syngap1-epilepsy-ugcPost-7457791427486466048-j8SB

 

WEBINARS!

CURE-ID Webinar tomorrow! Register now. Thu May 7, 2026 1:30pm – 3pm (PDT) cureSYNGAP1.org/cureID 

 

Next week, especially NY families, while you are registering, please make sure to go to this one with Dr. Buxbaum from Mt. Sinai. curesyngap1.org/seaver https://www.linkedin.com/posts/curesyngap1_curesyngap1-syngap1-rarediseaseresearch-activity-7457507565527044096-dDEK

 

Longitudinal Data Matters - Every six months or more!

 

Study list!

• CURE SYNGAP1 CONNECT http://curesyngap1.org/connect

• Citizen Health https://www.citizen.health/ai-advocate/syngap1

• Combined Brain

• ProMMiS https://www.linkedin.com/feed/update/urn:li:activity:7450196488300728320 & Rare-X, the same week.

• OR DSC. #S10e

• CURE-ID for Drug responses. Webinar: Thu May 7, 2026 1:30pm – 3pm (PDT) cureSYNGAP1.org/cureID

 

6th ANNUAL SPRINT FOR SYNGAP1, we raised over $300k! (Gross)

Thank you Tavillas for raising over $170k this will go to the Missense fund. We spent over $¼M on missense last year.

 

Thank you Emily Barnes for hosting a New England event for families. https://www.linkedin.com/posts/ecarlisle_we-had-an-incredible-sprint-for-syngap-ugcPost-7457845816502845440-nJCd

 

Thank you to Sara and Sarah for the Virginia event.  GREAT WORK. https://www.linkedin.com/posts/sarah-sakly-648544337_syngap1-raredisease-curesyngap1-ugcPost-7456869206341464065-VESz

 

Thank your Rifton for the donation of the tricycle.  Congratulations Matthew! https://www.linkedin.com/posts/curesyngap1_syngap1-curesyngap1-sprint4syngap-activity-7457806850676281344--JR-

 

Thank you to the Edouard Family! cureSYNGAP1.org/Crafts26

https://www.linkedin.com/posts/curesyngap1_curesyngap1-syngap1-craftsforacure-activity-7457211260242411520-ekPz

 

INAUGURAL SF NIGHT OF IMPACT, CA – 22 days

Join us this is our only Gala for 2026!

cureSYNGAP1.org/SF26

 

5TH SCRAMBLE FOR SYNGAP, SC – 150 days

Classic case of a small event becoming an institution!

cureSYNGAP1.org/Scramble26

 

USA: use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142

 

PUBMED

Pubmed 2026 is at 27. +10 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date

 

SOCIAL MATTERS

4,931 LinkedIn.  https://www.linkedin.com/company/curesyngap1

1.56k YouTube.  https://www.youtube.com/@CureSYNGAP1

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

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

 

$CAMP closed at $3.97 Friday.

https://www.google.com/finance/beta/quote/CAMP:NASDAQ

 

Like and subscribe to this podcast wherever you listen. https://curesyngap1.org/podcasts/syngap10

Episode 206 of #Syngap10 #CureSYNGAP1 #Podcast

Monday, April 20, 2026 - Week 17

 

CURE SYNGAP1 joins the Haystack Project in petitioning FDA for more clarity.

PR: https://static1.squarespace.com/static/5966cc2220099e91326caaec/t/69d7dbb80155f46e144ae2e5/1775754168227/4.9.26+press+release+petition_vf.pdf

Petition: https://static1.squarespace.com/static/5966cc2220099e91326caaec/t/69d7e016d5986e16ff09b64c/1775755288463/Letter+Head+Petition+for+rulemaking+to+amend%C2%A021+CFR+%C2%A7+314.126+and+%C2%A0%C2%A7312.47+%281%29.pdf

Reuters 4/1: https://www.reuters.com/sustainability/boards-policy-regulation/rare-disease-advocacy-group-urges-trump-administration-restore-fda-clarity-2026-04-01/

Pink Sheet: https://insights.citeline.com/pink-sheet/pathways-and-standards/review-pathways/could-structured-not-ad-hoc-us-fda-flexibility-increase-rare-disease-development-certainty-BYXJENIJLFEINOSO72RS53ZJHE/

 

Show your support here: https://www.regulations.gov/document/FDA-2026-P-3666-0001

Paragraph 1 – Share information about you and SYNGAP1.

Paragraph 2 – Seizures are hard to count while X, Y and Z are major burdens but FDA wants nice countable seizures, this makes it hard to develop drugs.

Paragraph 3 – How could “clinically meaningful” endpoints potentially help your community and drug developers? How could a study design other than a ‘randomized clinical trial’ help? How could FDA consulting with disease-specific experts help?

Closing  – Finish your letter with anything along these lines: We support the framework for all rare set diseases in Haystack’s petition. We don’t believe FDA has to lower the evidentiary bar to approve treatments for our diseases. Randomization isn’t always possible. New scientific methods should be considered. Endpoints specific to our disease should be considered. We urge FDA to open a rulemaking so we can have a legally binding regulation.

 

Board changes, thank you to everyone. https://www.linkedin.com/posts/curesyngap1_curesyngap1-syngap1-patientadvocacy-activity-7450528611339616256-4MJF?utm_source=share&utm_medium=member_desktop&rcm=ACoAAAAD8f4B7JC4TMss45Q8hrsq5kiceI0Z8HE

Press Release cureSYNGAP1.org/PR45

 

US, use your ICD-10, F78.A1: https://onlinelibrary.wiley.com/doi/10.1002/epi.70142

 

Study list!

• Citizen Health https://www.citizen.health/ai-advocate/syngap1

• Combined Brain (May 1 & 2 in NorCal), https://docs.google.com/presentation/d/1IjaHILXj7AlBDlbTJgvYrkBS_0bnI8VCnTIiPXJ7JGM/edit?usp=sharing 

• ProMMiS https://www.linkedin.com/feed/update/urn:li:activity:7450196488300728320 

• Rare-X, the same week.

• DSC and Cook’s are coming soon!

• CURE-ID for Drug responses.

 

CURE-ID is cool. https://cure.ncats.io/home (Webinar coming)

Webinar: Thu May 7, 2026 1:30pm – 3pm (PDT) cureSYNGAP1.org/cureID 

 

6th ANNUAL SPRINT FOR SYNGAP1, EVERYWHERE – 5 days - $207k! Go Tavilla.

17 teams raised $265K last year; this year, we have 20+ teams!

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

Thank your Rifton for the donation of the tricycle.

Email today: https://mailchi.mp/curesyngap1.org/sprint-for-syngap-2026-one-community-one-goal?e=17610baa03

 

INAUGURAL SF NIGHT OF IMPACT, CA – 38 days

Join us this is our only Gala for 2026!

cureSYNGAP1.org/SF26

 

5TH SCRAMBLE FOR SYNGAP, SC – 166 days 

Classic case of a small event becoming an institution!

cureSYNGAP1.org/Scramble26

PUBMED

Pubmed 2026 is at 26. +9 vs the week. (61 last year was +9) https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2026-2026&sort=date

 

SOCIAL MATTERS

4,891 LinkedIn.  https://www.linkedin.com/company/curesyngap1

1.55k YouTube.  https://www.youtube.com/@CureSYNGAP1

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

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

 

$CAMP closed at $4.67 Friday.

https://www.google.com/finance/beta/quote/CAMP:NASDAQ

 

Like and subscribe to this podcast wherever you listen.  https://curesyngap1.org/podcasts/syngap10

Episode 205 of #Syngap10 #CureSYNGAP1 #Podcast