August 23, 2025

GALA is now, #StokedAboutStoke, Go Grann, Our Villages, CHOP/NIH, More on Elopement. #S10e179

19 minutes

Friday, August 22nd, 2025. Week 34.

The 5th Annual Gala is happening now!

https://www.linkedin.com/posts/curesyngap1_syngap1-curesyngap1-galaforsyngap1-activity-7363593302312402944-W_TZ

cureSYNGAP1.org/Gala5

Sad to miss it? Join us in Boston or South Carolina.

Beacon of Hope September 12, 2025 - Boston, MA cureSYNGAP1.org/Beacon25

Scramble for SYNGAP October 4, 2025 - Greer, SC cureSYNGAP1.org/Scramble

Stoke Therapeutics indicates they will have a target for SYNGAP-1 in 2026!

https://investor.stoketherapeutics.com/news-releases/news-release-details/stoke-therapeutics-reports-second-quarter-2025-financial-results

12 Aug 2025 “Lead optimization is underway to identify a clinical candidate for the treatment of SYNGAP-1 in 2026. SYNGAP-1 is a severe and rare genetic neurodevelopmental disease.”

Just over 20 FDA approved Oligos and siRNAs today. We are still so early.

https://www.advancingrna.com/doc/moving-beyond-solid-phase-synthesis-the-momentum-of-oligonucleotide-manufacturing-0001

Congrats to Monica E. & Grann Therapeutics, seeing a child dosed for the first time with a novel medicine was remarkable.

https://www.grannpharma.com/press-releases

The SYNGAP1 Village: How Extended Family Can Provide Vital Support

https://curesyngap1.org/blog/syngap1-village-extended-family-can-provide-support/

Here’s a fun topic to discuss with your family, brain donation. https://www.autismbrainnet.org/

55yo with Dravet, lots of insights, Brava to Dr. Andrade and team! https://onlinelibrary.wiley.com/doi/10.1111/epi.18613

SRF joins with CHOP, Wistar and other Philly-area research institutions with a letter to urge legislators to reject NIH cuts.

8/20/25 Letter can be viewed in SRF Public-facing drive

https://drive.google.com/file/d/1HHmCAuRYAQxb_1DtMtkQTz3H8__g9zKq/view?usp=drive_link

Philadelphia Inquirer picked up the story 8/20/25

https://www.inquirer.com/health/medical-research-institutions-reject-nih-cuts-20250820.html

More on #Elopement: Alarms, Roofs, Resonated. Keep talking to doctors about this. Post is up to 139 Votes, percentages little changed, join the conversation on FB. https://www.facebook.com/groups/syngap/posts/1734514154096968/

#S10e178 - https://www.youtube.com/watch?v=OiRnXxh0wfY

Conference is in 103 Days

https://curesyngap1.org/events/conferences/cure-syngap1-conference-2025-hosted-by-srf/

Pubmed is at 38!

https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&filter=years.2025-2025&timeline=expanded&sort=date&sort_order=asc

SHARE BLOOD TO THE SRF BIOBANK AT CB!

https://curesyngap1.org/blog/fueling-research-syngap1-combinedbrain-biorepository-roadshow/

VOLUNTEER

Join us: https://curesyngap1.org/volunteer-with-srf/

SOCIAL MATTERS

- 4,285 LinkedIn. https://www.linkedin.com/company/curesyngap1/

- 1,420 YouTube. https://www.youtube.com/@CureSYNGAP1

- 11,294 Twitter https://twitter.com/cureSYNGAP1

- 46k Insta https://www.instagram.com/curesyngap1/

NEWLY DIAGNOSED?

Next New Family Webinar - Tuesday Sept. 9th, 2025, 5 PM Pacific scheduled! https://curesyngap1.org/resources/webinars/webinar-105-syngap-research-fund-quarterly-webinar-new-syngap1-family-orientation/

Resources

https://curesyngap1.org/syngap1-resources-for-newly-diagnosed-families

Podcasts, give all of these a five star review!

https://cureSYNGAP1.org/SRFApple

https://podcasts.apple.com/us/channel/syngap1-podcasts-by-srf/id6464522917

Episode 179 of #Syngap10

#Advocate #PatientAdvocacy #UnmetNeed #SYNGAP1 #SynGAP #SynGAProMMiS

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By Syngap Research Fund, 501(c)(3)

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