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 ...
Solid Biosciences Inc. (NASDAQ:SLDB) is one of the stocks that will double in 2026. On December 4, Needham analyst Gil Blum ...
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 ...
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.
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 ...
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