A University of California San Diego-led team has discovered that restoring a key cardiac protein called connexin‑43 in a ...
Jan 26 (Reuters) - Sarepta Therapeutics' gene therapy for a progressive muscle‑wasting disorder showed sustained benefit in ...
InvestorsHub on MSN
Sarepta posts encouraging three-year data for Duchenne gene therapy
Sarepta Therapeutics Inc. (NASDAQ:SRPT) released three-year follow-up results from its EMBARK study, showing that its gene ...
Investor's Business Daily on MSN
Sarepta pops on Elevidys data; but will it save the embattled gene therapy?
Sarepta stock jumped early Monday after the company unveiled three-year test results for its embattled gene therapy, Elevidys ...
A new CRISPR-based one-off procedure that lowers "bad" cholesterol has been approved to enter Phase I human trial. If ...
Under the agreement, participating states will receive “discounts and rebates” from the drugmakers if the treatments don’t ...
GlobalData on MSN
Genespire to take rare disease gene therapy to clinic in 2026
GENE202 is a single dose gene therapy for patients with rare metabolic disease, methylmalonic acidaemia.
Precision DNA editing gene therapy achieves deep remissions in aggressive leukemia previously considered incurable.
Orphan Drug Designation applies to approximately 50,000 Patients who predominantly suffer from severe chronic pain due ...
Doctors have delivered gene therapy during heart bypass surgery for the first time, aiming to strengthen blood vessels ...
The Integrated Health Clinic at University Medical Center New Orleans with LSU Health New Orleans is one of only six teams ...
"Regulatory flexibility must be tailored for cell and gene therapies," commented FDA Commissioner Marty Makary. "These are ...
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