qjgfkuwilliams.bsky.social
👤 Joined on 05 May 2026
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@qjgfkuwilliams.bsky.socialAnother 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.
Show PostSarepta Therapeutics Announces Presentations at 2026 World Muscle Society Annual Congress www.stocktitan.net/news/SRPT/sa...
Show PostRNA 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.
Show PostA 30-day insurer deadline for FDA-approved specialist-prescribed treatments is exactly what DMD families need because no family should require a foundation to win an insurance appeal for Elevidys while their son loses muscle function waiting on bureaucracy that moves slower than disease.
Show PostWhat 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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