PLAY PODCASTS
AAV.  Resources on cureSYNGAP1.org.  Congrats to Ultragenyx & Novartis. Conf is in 5 days! #S10e190
Episode 191

AAV. Resources on cureSYNGAP1.org. Congrats to Ultragenyx & Novartis. Conf is in 5 days! #S10e190

CURE SYNGAP1 PODCAST aka SYNGAP10

November 27, 20259m 57s

Audio is streamed directly from the publisher (mcdn.podbean.com) as published in their RSS feed. Play Podcasts does not host this file. Rights-holders can request removal through the copyright & takedown page.

Show Notes

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