CAMP4, JAX and Allen present about ASCGT, JHU Article, CRISPR, Repurposing, 4PB & SRF News #S10e172
Description
Sunday May 18, 2025. Week 21
Show notes on the site: https://curesyngap1.org/podcasts/syngap10/
CAMP4
Press Release https://investors.camp4tx.com/news-releases/news-release-details/camp4-presents-translational-data-syngap1-related-disorders
Dr. Yuri at WODC: https://www.youtube.com/watch?v=oNwgo7TmrVo
Presentation at ASGCT: https://investors.camp4tx.com/news-events/presentations
To get these Therapies to kids the NHS data is key, ProMMis
CHOP - Email them ENDD@chop.edu
Stanford (Singed!) - https://curesyngap1.org/blog/srf-announces-stanford-launches-california-synaptopathy-clinic/
Citizen Health
https://cureSYNGAP1.org/Citizen is at 267, why not 300?
Hopkins article featuring SRF - Competition is good for the patient
LI
ASGCT
- Mouse poster for Q504X from the JAX, Dr. Matt Simon
- Conf Video https://www.youtube.com/watch?v=loYXkkTSUIY
- Presentation on AAV for SYNGAP1 at Allen by Dr. Megan Quinlan
What about this guy?
PBS: https://www.youtube.com/watch?v=dOeiPoa3gvM
Eric Topol: https://erictopol.substack.com/p/the-first-human-to-undergo-in-vivo
More incredible news
https://news.unl.edu/article/husker-team-wins-second-round-prize-in-nih-gene-editing-challenge
REPURPOSING
Mike’s post in private SRF Group: https://www.facebook.com/groups/syngap
#Amlexanox with Fortuity Pharma. https://fortuitypharma.com/
Butyrate Update
Trial ending at year end, working with SLC6A1 and Rachel Heilmann to continue coverage, then we can scale, once we have a paper.
SunButyrate https://a.co/d/7kiHXS8
PUBMED
328 total with 20 YTD
https://pubmed.ncbi.nlm.nih.gov/?term=syngap1&sort=date&sort_order=asc&timeline=expanded
Sleep https://www.sciencedirect.com/science/article/abs/pii/S1389945724005793?via%3Dihub
SRF Fundraisers
MDBR with AJH and HM https://curesyngap1.org/calendar/million-dollar-bike-ride-2025/
Well done to Tavilla and Jones! We are over $221k
Tavilla clears $160k for Sprint.
https://donate.curesyngap1.org/campaign/sprint4syngap2025/c660255
Porter Jones and his friends William, Patrick, and Judson raised over $10k in doughnuts, this netted over $8k because people donated. Amazing and thank you to Krispy Kreme.
https://donate.curesyngap1.org/event/sweets-for-syngap1/e678677
SRF PROFILES
Vicky post in Mexico https://www.linkedin.com/posts/victoria-arteaga-26913433_diagnostico-epilepsia-syngap1-activity-7329725268003069953-R41P
Vicky’s Siblings Violeta and Lourdes, sisters to Amelia https://curesyngap1.org/syngap-siblings/
Harper Albrecht Wednesday Warrior #215 parents Ashley and Justin https://cureSYNGAP1.org/Warrior
PREREGISTER FOR THE CURE SYNGAP1 CONFERENCE
https://curesyngap1.org/calendar/cure-syngap1-conference-2025-hosted-by-srf/
United States
