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 ...

Doug Ingram of Sarepta Therapeutics is my pick for the Worst Biopharma CEO of 2025. He was an easy selection, but unlike some ...

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

(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 ...

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 ...

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 ...

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 company said in a statement that the FDA notified it on Monday and plans to resume shipping Elevidys to sites of care for treatment of ambulatory patients with Duchenne “imminently. “ The FDA ...

New protocol adds a second immunosuppressant to the regimen of patients who are at higher risk for liver complications Children’s Hospital Los Angeles (CHLA) is expanding its safety protocol for ...

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.

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 ...