By Mariam Sunny Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for Duchenne muscular dystrophy slowed disease ...
After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing ...
Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...
Researchers complete final analysis of a phase 3 study of men with hemophilia B receiving intravenous etranacogene dezaparvovec.
Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...
Precision DNA editing gene therapy achieves deep remissions in aggressive leukemia previously considered incurable.
Medicaid is doing a novel payment system for the new, promising and expensive sickle cell treatment. It may become a model ...
Universal gene‑edited CAR‑T therapies turn donor T cells into off‑the‑shelf cancer treatments, cutting delays and costs while ...
A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...
University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a mouse ...
On a Monday conference call, Sarepta CEO Doug Ingram said new long-term data should help “rebalance the discussion” ...
Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for a muscle‑wasting disorder slowed disease progression and showed ...
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