Sarepta Advances Precision Genetic Therapies Toward Full FDA Approval: AMONDYS 45 and VYONDYS 53 Seek Traditional Approval Status
- April 20, 2026
Simple Daily Habits to Boost Your Whole-Body Health and Well-Being
Extended Acoramidis Data Show Durable Survival Advantage in ATTR-CM
New Study Reports How Individual Areas of the Brain Age
LISTEN TO OUR PODCAST
THE LATEST FROM PATIENT WORTHY
FEATURED
UPCOMING EVENTS
SIGN UP FOR OUR NEWSLETTER
SMA treatment and management involves a combo of advanced medical therapies and ongoing supportive care. From targeting the genetic cause to supporting everyday function, a comprehensive approach can make a meaningful difference in quality of life. Click the link to share your story today! bit.ly/3VuxFDV #sma #GeneTherapy #PatientWorthy
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
