Trimethylaminuria
My Journey, My Life, My Story, My Book – Carry’s Experiences with “Fish Odor Syndrome”
You can not see, hear, or touch my rare condition. I STINK! (LITERALLY) I wasn’t sure if I was going to tell my story. But
Extended Acoramidis Data Show Durable Survival Advantage in ATTR-CM
New Study Reports How Individual Areas of the Brain Age
Optimized Dosing Delivers Enhanced Motor Benefits: FDA Approves High-Dose Spinraza Regimen for Spinal Muscular Atrophy
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Understanding how SMA is tested is a key step toward earlier diagnosis and faster access to care. From genetic blood testing to newborn screening, modern tools are helping identify spinal muscular atrophy sooner and more accurately than ever before. #spinalmuscularatrophy #genetictesting #PatientWorthy
International Foundation for CDKL5 Research-IFCRHave you registered for the CDKL5 Family Conference yet? You won’t want to miss this.👇
We’re hosting a powerful, honest session on gene therapy - starting with real talk about where the science stands, what risks exist, and what expectations are realistic.
Then we’ll move into a community risk/benefit workshop where every voice matters. Whether you’re eager to join a trial, unsure, or not interested at all, we need your perspective to understand the full picture of our community’s hopes and concerns.
Your voice shapes the future - We hope to see you there!! 💜✨
#Conference2026 #GeneTherapy #EducationalFamilyConference #RareDiseaseCommunity #CDKL5 #IFCRCommunity
United Mitochondrial Disease FoundationLooking for connection and support? Join patients and families from the mito community at one of UMDF’s virtual Support Meetings. Visit our events calendar for login details and come be part of the conversation. umdf.org/events-calendar/
