Werner is CEO of Alltrna and CEO-partner at Flagship Pioneering. Duchenne muscular dystrophy has seen more progress in the past 15 months (give or take) than in the last couple of decades combined. In ...

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 ...

From Philly and the Pa. suburbs to South Jersey and Delaware, what would you like WHYY News to cover? Let us know! Yuva Gambhir has a full life. The University of Pennsylvania senior studies cognitive ...

Prior to initiating treatment, baseline platelet counts and triglycerides should be obtained. Duvyzat ™ (givinostat) oral suspension is now available for the treatment of patients 6 years of age and ...

Patients with Duchenne muscular dystrophy now have new hope with gene therapy. And for an Illinois family, it’s a double blessing. On their family farm in rural Illinois, the Flessner boys have free ...

Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to disease-related loss of muscle and movement, adverse effects of ...

Like most mothers, I only saw perfection when my first son was born. That quickly changed when we began to question pediatricians about his physical and cognitive delays. Don’t worry, they said. Boys ...

INDIANAPOLIS — A local mom is pushing for a rare and deadly disease to be added to Indiana's newborn screening panel. Duchenne muscular dystrophy affects at least 1 in 5,000 male births each year.

At Sarepta Therapeutics, we’ve seen it all. Here are the questions I believe we should be asking to move forward in Duchenne muscular dystrophy.

A drug infusion is hardly dramatic, especially for a kid like 12-year-old Brecken Kinney, who has been poked and prodded his entire life to treat his Duchenne muscular dystrophy. But the fluid pumping ...

Logan Farley brought his bait, pole, and his patience to his favorite fishing spot. He's faced a lot challenges, and life's tested him far beyond the fishing hole. Stephanie and Patrick Farley saw a ...