Grilled Cheese

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@dingelldonel.bsky.socialSep 17, 2026, 6:48 PM

"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

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