About 500 children worldwide have received the gene therapy, including some families traveling abroad to gain access to it.

The Killian family, of Rockwall, are celebrating a record-breaking fundraising milestone with their annual Sam's Night event, ...

Months after disclosing significant layoffs across its North Carolina operations, Pfizer (NYSE: PFE) plans to eliminate more ...

Pfizer has told 75 employees in Sanford, North Carolina, that their positions have been eliminated. | Pfizer has told 75 ...

Jefferies initiates buy coverage on Sarepta Therapeutics (SRPT) due to Duchenne muscular dystrophy franchise and strong ...

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

Pfizer suffered a blow earlier this month when new data showed that its gene therapy for Duchenne muscular dystrophy failed ...

While success is far from guaranteed in bio-land, with this week’s raising, Percheron has the horsepower to have a decent ...

Parents of 25-year-old Mats Steen, who sadly died in 2014, discovered that their son had a secret life online.

A Pfizer result is unsettling the gene therapy push for Duchenne muscular dystrophy: The boys didn't improve even though a ...

SAVANNAH, Georgia — Heart problems are common in the vast majority of neuromuscular disorders, and cardiac monitoring of ...

Robert and Trude Steen assumed their late son led ‘a wasted life’ – until the discovery of his alternate identity as a ...