In Duchenne muscular dystrophy (DMD), cardiomyopathy, cardiac conduction abnormalities, or both usually appear in patients by age 10. “It’s important to know that it’s probably present to ...

Opens in a new tab or window SAVANNAH, Ga. -- Delandistrogene moxeparvovec (Elevidys), a gene therapy approved to treat Duchenne muscular dystrophy, missed its primary endpoint but differences ...

A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular ...

Pfizer is “evaluating appropriate next steps” for its Duchenne muscular dystrophy (DMD ... patient in the phase 2 DAYLIGHT study died of a cardiac arrest after receiving the one-shot treatment ...

Sarepta Therapeutics has ended development of a late-phase Duchenne muscular dystrophy (DMD) drug candidate over a safety problem. The safety signal deprived Sarepta of a near-term shot at accelerated ...

Capricor Therapeutics' bid to find a partner for its Duchenne muscular dystrophy (DMD ... including skeletal and cardiac endpoints, according to Capricor, which presented the results at the ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...

People with Myotonic Dystrophy Type 1 (DM1), the most common adult-onset form of muscular dystrophy, progressively lose ...

Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company, announced today that the United States (U.S.) Food and Drug Administration (FDA) granted Orphan Drug Designation and ...

PTC Therapeutics' NDA resubmission of Translarna was based on data from Study 041 and the long-term STRIDE registry. No PDUFA action date was provided for regulators.

Edgewise Therapeutics, Inc. ( (EWTX) ) has released its Q3 earnings. Here is a breakdown of the information Edgewise Therapeutics, Inc.

Nashville, Tennessee Friday, November 8, 2024, 13:00 Hrs [IST] ...