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.org/podcasts/syngap1-stories/ 

A gold mine have you listened to #38, the Virginie Pod, really must listen, she is our leader. https://www.linkedin.com/posts/graglia_syngap1stories-syngap1-syngap1storiesty-activity-7387203351907708928-liNL 

CLINICAL TRIAL & GENETIC MEDICINE CORNER

Example of Ultragenyx FAST Angelman follow on trial to look at other ages and genotypes, key message, never give up. https://www.linkedin.com/posts/cureangelman_the-global-aurora-study-will-enroll-approximately-activity-7389647402690957312-Bihi

Congrats to Novartis on approval of the first Gene Therapy to Cure SMA!

https://www.linkedin.com/posts/graglia_sma-fdaapproval-rarediseaseinnovation-activity-7398939783005347840-Ocd_ Remember Spinraza was approved in December 2019.

TODOS

1. Sign up for Citizen Health: https://www.citizen.health/partners/srf
2. USE YOUR ICD-10 F78.A1 #S10e185 https://www.youtube.com/watch?v=dale0NbxDpU
3. Go to CURE SYNGAP1 Conference 2025 Atlanta: https://curesyngap1.org/events/conferences/cure-syngap1-conference-2025-hosted-by-srf/

SOCIAL MATTERS

4,468 LinkedIn.  https://www.linkedin.com/company/curesyngap1/ 

1,480 YouTube.  https://www.youtube.com/@CureSYNGAP1   

11.2k Twitter https://twitter.com/cureSYNGAP1 

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

$CAMP stock is at $3.62 on 26 Nov. ‘25 https://www.google.com/finance/beta/quote/CAMP:NASDAQ

Episode 190 of #Syngap10 #CureSYNGAP1