Rare diseases, exceptional people: Filippo Buccella has battled for 25 years to improve care for children with Duchenne Muscular Dystrophy (DMD ... Doctors offered little hope for his future ...

As a child, Mats had been diagnosed with Duchenne muscular dystrophy, a genetic disorder ... But as in any media or art form, the greatest stories tend to be ones that reflect our lives back ...

28, 2024 — Using state-of-the-art technology ... lead to new treatments for people with Duchenne ... New Gene Therapy for Muscular Dystrophy Offers Hope July 17, 2024 — A new gene therapy ...

Patients with Duchenne muscular dystrophy (DMD) have long been without effective treatment ... inflammation in muscles and eventually atrophy or reduced muscle mass. The hope is that the drug will ...