Friday, April 25, 2025 – DNA Day
Good luck at Sprint tomorrow. $194k at this moment.
WODC Readout
Saw friends (Hebbian Bio, Unravel, Ana M, Balaji) and families (HM AN E)
RH Drug talk – https://www.youtube.com/watch?v=Hpj72nMFfZc.
(Other Family Day Presentations from SYNGAP1 Conference in LA are on YouTube https://www.youtube.com/playlist?list=PLjpr3a14_ls2ummdbWyUdvRpMcQBlRXy2)
Read out in a panel where we had to really talk through small molecule (broad – LB) vs precision (narrow C4/STK) and broad (Tevard).
ASO Update: Trials need heterogenous populations. At first, I expect we will be looking for PTVs beyond the first 3 or 4 exons. Once approved, or in subsequent trials, I assume it will be available more broadly but not at first. Trials are coming, take notes and videos.
Press Release on Roadmap: https://www.linkedin.com/posts/graglia_progress-in-syngap1-therapeutic-development-activity-7321668845495066624-1dDn/
Oliver’s Warrior Story – https://curesyngap1.org/syngap-warriors/oliver/
Sign up for the conference: 222 Days! https://curesyngap1.org/events/conferences/cure-syngap1-conference-2025-hosted-by-srf/
Impact Report Webinar can be watched in its entirety as well as in segments – 18 of them to cover most of the topics discussed are being added to our YT channel at https://www.youtube.com/playlist?list=PLjpr3a14_ls3OvtdFahBr4wAa8BL7aUkv
Effie on TJB https://www.linkedin.com/posts/graglia_well-deserved-kudos-for-dr-terry-jo-bichell-activity-7321280877068541952-9f_1/
CB Blood Drive this weekend in TN. Read here for more information: https://curesyngap1.org/blog/fueling-research-syngap1-combinedbrain-biorepository-roadshow/
Newly Diagnosed Resources: https://curesyngap1.org/Resources