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 ...

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.

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 ...

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 ...

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 ...

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 ...

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 ...

The FDA now limits Elevidys to walking boys ages 4 and up after two teens died from liver failure The therapy will now carry a boxed warning for serious liver injury, liver failure and death Doctors ...

MONDAY, Nov. 17, 2025 (HealthDay News) — The U.S. Food and Drug Administration (FDA) is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died ...