Mitzie Coutts

abenespina887.bsky.social

16 October 2026 07:24

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 07:23

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.

hilalia.bsky.social

16 October 2026 07:21

What stood out to me is that the family is working directly with SRPT and Phoenix Children’s Hospital to fight for continued DMD treatment, because screening only matters if patients can actually access and stay on therapy. www.pinalcentral.com/opinion/loca...

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