A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would ...

A new class of life-saving drugs is helping children who once had no hope. But some carry a price tag of millions for a ...

(RTTNews) - The U.S. Food and Drug Administration is investigating the death of an 8-year-old boy who received Elevidys, a gene therapy developed by Sarepta Therapeutics for Duchenne muscular ...

WASHINGTON, D.C. — The U.S. Food & Drug Administration announced Friday that it has launched an investigation into the death of an 8‑year‑old boy who received Elevidys, the gene therapy developed by ...

CHMP issued a negative opinion on Elevidys for Duchenne patients aged 3–7 despite secondary gains in motor function. Elevidys has treated over 900 DMD patients, but EMA and FDA raise concerns over ...

The U.S. FDA said on Monday that it recommends lifting the voluntary hold on Sarepta Therapuetics' gene therapy, Elevidys, in ambulatory patients. Last Friday, the FDA said that it will investigate ...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA ...

The U.S. Food and Drug Administration said on Friday that it is investigating the death of an 8-year-old boy who received Elevidys, a Sarepta Therapeutics (NASDAQ:SRPT) gene therapy for Duchenne ...

Sarepta and Hansa formed the partnership in 2020 to determine if Idefirix could broaden the patient population for Elevidys.

The FDA said Nov. 14 it will place its most prominent safety warning on Elevidys, a gene therapy for Duchenne muscular dystrophy, after two fatal cases of acute liver injury. Officials have reported ...

Approximately 25 non-ambulatory participants will receive sirolimus as part of the regimen in Cohort 8 of the ENDEAVOR study, which is expected to begin before the end of the year The enhanced ...