Mitzie Coutts

abenespina887.bsky.social

16 October 2026 08:43

Another FDA setback for RGNX, and with the resubmission timeline already slipping, the focus now has to be on what the data and safety profile can actually support.

Christine Leola

dingelldonel.bsky.social

16 October 2026 08:42

Sarepta Therapeutics Announces Presentations at 2026 World Muscle Society Annual Congress www.stocktitan.net/news/SRPT/sa...

Christine Leola

dingelldonel.bsky.social

16 October 2026 08:40

“10 years ago, there was nothing and the prospect was scary.” Families refused to accept “nothing” as the answer, and today, therapies like elevidys are part of the path they fought to create. That’s why we all should keep pushing. 💙 www.youtube.com/watch?v=MhLZ...

Christine Leola

dingelldonel.bsky.social

16 October 2026 08:40

"False hope" is a bold take for a drug with a 90% real world continuation rate and a confirmatory study fully enrolled since 2023. C'mon. Let the data answer the clinical benefit question before the headlines write the verdict #RareDisease #DMD #AcceleratedApproval #LetTheScienceLead

Christine Leola

dingelldonel.bsky.social

16 October 2026 08:39

RNA therapy working in ALS is exactly the kind of proof of concept that validates what Sarepta's siRNA platform is building toward for FSHD, DM1, and Huntington's. The platform story keeps getting stronger.