Suneel Ram is a spiritual seeker, sings in a band and was the subject of a comic novel two years ago about living with Duchenne muscular dystrophy, a rare genetic ...

The family of two boys who both have a life-limiting condition are hoping to take a trip to Disneyland Paris later this year.

Patients in Scotland can't get the treatment - which is available in England - despite manufacturers giving it away for free ...

A federal advisory committee that provides guidance on which rare diseases newborns should be screened for has been dissolved ...

Generating significant returns and multiplying their money in the stock market remains a primary goal for most investors.

The firm said the clinical trial pause is temporary as an independent data monitoring committee finalizes its analysis for submission to regulators.

Therapeutics shared the following update related to ELEVIDYS, the only approved gene therapy in patients with Duchenne muscular ...

A pair of Scottsdale brothers have launched an online site to help address what they consider an oft-forgotten part of the ...

Some locals will be embarking on an ambitious journey down the Rogue River later this month for a good cause. A group of ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved ...

Healthcare Analysts, along with Dr. Yaacov Anziske of SUNY Downstate, discuss Sarepta’s (SRPT) ELEVIDYS and the future of Duchenne Muscular ...

BioMarin Pharmaceutical Inc. has advancements in phenylketonuria treatment. Click to learn how Palynziq's success could ...