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Over 1200 families are caring for a loved one with the rare disease ”SynGAP” resulting from a variant of the SYNGAP1 gene. This 10 minute weekly podcast is for them. A quick summary of the latest news in the space. The host is Mike Graglia, co-founder & managing director of the SynGAP Research Fund. SRF is a parent-led, all volunteer public charity in the US that strives to accelerate research into treatments for SYNGAP1 so that we can help our loved ones in a timeframe that matters. Learn more at https://www.syngapresearchfund.org/
Episodes
Friday Feb 28, 2025
Friday Feb 28, 2025
Friday, February 28, 2025 - Week 9
Hope vs. Despair?
Despair:
- NIH Cuts
NBC https://www.nbcnews.com/science/science-news/trumps-nih-budget-cuts-threaten-research-stirring-panic-rcna191744
NYT https://www.nytimes.com/2025/02/25/briefing/president-trump-vs-medical-research.html?unlocked_article_code=1.0U4.-7WW.F3yrk2tjpLJe&smid=url-share - FDA Staffing Cuts
https://www.biopharmadive.com/news/fda-layoffs-trump-doge-hhs-cuts-impact/740499/ - Medicaid Cuts https://www.disabilityscoop.com/2025/02/27/house-vote-tees-up-billions-in-cuts-to-medicaid-disability-services/31321/
https://www.politico.com/news/2025/02/27/republicans-medicaid-expansion-budget-00206612 - President who doesn’t care https://time.com/7002003/donald-trump-disabled-americans-all-in-the-family/
- DOGE head who also doesn’t care
Hope:
- Pipeline https://curesyngap1.org/syngap1-related-disorder-therapeutic-pipeline/
- Community https://curesyngap1.org/team/
- Global: https://syngapglobal.net/
- ICD-10 Code https://www.rareepilepsynetwork.org/about-icd-codes
Disability is Political
Take it or Advocate?
Taking it does not work.
Advocate:
- SRF https://curesyngap1.org/srf-legislative-advocacy-efforts-for-syngap1/
- ELF https://everylifefoundation.org/
- Research America
https://www.researchamerica.org/marys-letters/research-advocates-unite-in-force/ - Alliance for a Stronger FDA
https://www.strengthenfda.org/ - Modern Medicaid Alliance
https://modernmedicaid.org/about-the-alliance/
Annie Kennedy’s comments at the ELF Capitol Briefing: https://www.youtube.com/live/F6kfKsYCQYU?si=ihWOl7Lda7Steg3p&t=3391
Responsibility to those who cannot or will not be here. 10% of Americans. 10k diseases, all chronic. Childhood. Do not have FDA approved treatments, no cures. Staggering Financial Costs aka Toxicities. Efforts to date have allowed for interventions that may still matter. We have invested so much.
Episode 163 of #Syngap10
#Advocate #PatientAdvocacy #UnmetNeed #SYNGAP1
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