Solid Biosciences Inc. (NASDAQ:SLDB) develops therapies for neuromuscular and cardiac diseases in the US. In addition, the ...

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 ...

Although the research centered on mAChR4 within the gut, this signaling protein is also known to influence areas of the brain involved in habit formation, learning and addiction. Because individuals ...

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

For the scientific enterprise, 2025 was marked by setbacks and challenges. But scientists are no strangers to adversity—the ability to overcome obstacles is built into our training. In turn, research ...

Capricor Therapeutics’ stock has soared by more than 370% after a pivotal trial of its Duchenne muscular dystrophy (DMD) cell ...

Acute liver injury has been associated with the death of two non-ambulatory DMD patients treated with Elevidys.

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 ...

NEW YORK – Sarepta Therapeutics on Tuesday released details on its plans to evaluate a new immunosuppressive regimen for treating non-ambulatory Duchenne muscular dystrophy patients with its gene ...

Sarepta Therapeutics, Inc. (SRPT) on Tuesday said that the U.S. Food and Drug Administration (FDA) has approved dosing in a study cohort to evaluate the use of an immunosuppressive regimen as part of ...

The U.S. Food and Drug Administration (FDA) on Tuesday approved dosing in Sarepta Therapeutics Inc.’s (NASDAQ: SRPT) Cohort 8 of ENDEAVOR (Study 9001-103). • SRPT shares are advancing steadily. Get ...

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 ...