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 ...

A mom was overjoyed when she learned a gene therapy treatment could save her child's life, until she found out no one would cover the $2 million price tag.

Sarepta Therapeutics SRPT announced that the FDA has approved significant changes to the label of Elevidys, its one-shot gene therapy for Duchenne muscular dystrophy (DMD). The revised labeling not ...

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 ...

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 ...

Wedbush upgrades Sarepta Therapeutics, citing overdone selloff and potential upside as FDA could re-approval Elevidys for non ...

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 ...

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) has approved dosing in Cohort 8 ...

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