SYNGAP10 is a 10 minute weekly blog to keep parents and families up to date on what the team at SynGAP Research Fund (SRF) is doing to advocate for patients& advance research into SYNGAP1. If you do nothing else, listen to or watch this and let us know what you think.
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SYNGAP10 is a 10 minute weekly blog to keep parents and families up to date on what the team at SynGAP Research Fund (SRF) is doing to advocate for patients& advance research into SYNGAP1. If you do nothing else, listen to or watch this and let us know what you think.
CAMP4, JAX and Allen present about ASCGT, JHU Article, CRISPR, Repurposing, 4PB & SRF News #S10e172
CURE SYNGAP1 PODCAST aka SYNGAP10
43 minutes 42 seconds
6 months ago
CAMP4, JAX and Allen present about ASCGT, JHU Article, CRISPR, Repurposing, 4PB & SRF News #S10e172
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
GETA Talk: https://www.facebook.com/Syngapresearch/posts/pfbid08Aztex32vdwNZduVkPQeG353W49Dhq8iKJeZEkajKUmkeUiDdCwzyqa9ndEVpdCUl
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/
CHCO - https://curesyngap1.org/blog/visiting-syngap1-natural-history-study-childrens-hospital-colorado-chco/
Citizen Health
https://cureSYNGAP1.org/Citizen is at 267, why not 300?
Hopkins article featuring SRF - Competition is good for the patient
FB https://www.facebook.com/cureSYNGAP1/posts/pfbid07MP7St3zdxLKYXTJULKa5S35YvgL6AJndenHicedxU3rtQF6iY1wwwNUoDuUD9cpl
LI
https://www.linkedin.com/posts/curesyngap1_nih-cuts-stalling-progress-on-rare-genetic-activity-7328449817074946048-AAsB
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?
Gift link: https://www.nytimes.com/2025/05/15/health/gene-editing-personalized-rare-disorders.html?unlocked_article_code=1.H08.E-oX.t5bo6kxJGUOU&smid=url-share
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
https://aesnet.org/abstractslisting/phenylbutyrate-for-syngap1-related-developmental-and-epileptic-encephalopathy
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/
CURE SYNGAP1 PODCAST aka SYNGAP10
SYNGAP10 is a 10 minute weekly blog to keep parents and families up to date on what the team at SynGAP Research Fund (SRF) is doing to advocate for patients& advance research into SYNGAP1. If you do nothing else, listen to or watch this and let us know what you think.
