Duchenne muscular dystrophy

sarepta building
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Sarepta, despite key trial flop, will seek full nod for DMD meds

Sarepta’s Vyondys 53 and Amondys 45 failed to show a statistically significant difference versus placebo on a key measurement of DMD patient mobility.
Angus Liu Nov 4, 2025 7:56am
Goal disorientation and lack of vision

UPDATED: Taiho’s discontinues ph. 3 trial after DMD miss

Jul 8, 2025 10:17am
Redbrick building with Sarepta name and logo
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2nd patient dies after Sarepta's DMD gene therapy Elevidys

Jun 16, 2025 10:58am
The Top Line

What were the biggest clinical trial flops of 2024?

Feb 21, 2025 9:00am
rocket ship blast off fire away take off

Solid soars as early DMD gene therapy data beat expectations

Feb 18, 2025 8:10am
DNA gene therapy gene editing helix CRISPR

Regenxbio starts pivotal study for DMD gene therapy

Nov 18, 2024 2:24pm