muscular dystrophy

Sarepta faces new scrutiny after 3rd patient death

Analysts grilled Sarepta on Friday for failing to disclose a third gene therapy patient death prior to a restructuring announcement earlier this week.

Fraiser Kansteiner Jul 18, 2025 10:19am

Edgewise drug lowers biomarker in ph. 2 muscular dystrophy trial

Dec 16, 2024 7:40am

DMD gene therapy uses cells to stitch together mRNA in mice

Nov 14, 2024 3:10pm

Avidity therapy shows possible benefit in muscular dystrophy

Jun 12, 2024 7:00am

pay deal hand over cash licensing transaction money

Sanofi pays Fulcrum $80M for rights to phase 3 rare disease drug

May 13, 2024 7:09am

PepGen gains a little pep in its step as FDA clears trial hold

Oct 12, 2023 7:00am