After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing.
Sarepta Therapeutics (NASDAQ:SRPT) reported three-year top-line results from its EMBARK Phase 3 pivotal trial evaluating ...
Sarepta Therapeutics (NasdaqGS:SRPT) reported positive three year functional results from its Phase 3 EMBARK trial of ...
Sarepta Therapeutics SRPT announced positive three-year top-line data from Part 1 of the phase III EMBARK study, evaluating ...
Dyne Therapeutics leverages its FORCE platform to target genetically driven muscle diseases. Read why I am bullish about DYN ...
In his CEO Commentary, Frederic Revah, Ph.D., underscores the dedication, expertise and successes of Genethon's 240 scientists and professional staff as they pursue new gene therapy treatments for ...
Some 200 rare disease therapies are at risk of losing eligibility for a pediatric priority review voucher, a recent analysis ...
The move can significantly lower out‑of‑pocket burdens for families, given that India’s health insurance coverage for ...
Quadriplegic and bedridden in a prefabricated home, 36-year-old Li Xia can only move one finger and one toe -- yet he runs a ...
KROS is advancing KER-065 for Duchenne muscular dystrophy, with orphan drug status and a phase II trial planned for early ...
Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease progression and showed sustained benefit in patients' ability to control and coordinate movement three years after ...
Dr. Mackey’s project, “ Regenerating healthy human skeletal muscle at single nucleus resolution ,” will create the most detailed molecular map to date of how healthy human muscle repairs itself. The ...
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