Scientists have developed a novel approach of genome editing to repair muscle stem (satellite) cells, offering new hope for ...

The US Food and Drug Administration has given the green light for the first gene therapy that treats a rare form of muscular dystrophy to be used in most people who have the disease and a certain ...

A Community Event for Duchenne Muscular Dystrophy Awareness Brick, United States – January 1, 2026 / Steps for Hope 5K / The ...

—Older age, presence of scoliosis, and other factors were associated with lower predicted forced vital capacity in patients with Duchenne muscular dystrophy using non-invasive ventilation. Individuals ...

The Food and Drug Administration (FDA) has approved the first gene therapy for the treatment of Duchenne muscular dystrophy (DMD) in children from age 4 through 5 years of age. Pediatric patients who ...

Scientists are testing nearly two dozen treatments that might stop the disease. But enrollment in the trials is very restricted, and few children qualify. By Gina Kolata Lucas was 5 before his parents ...

Editor’s note: This is an automatically generated transcript. Please notify [email protected] if there are concerns regarding accuracy of the transcription. Muscular dystrophy, as a category, is quite ...

When Elie and Nancy Eid founded Save Our Sons Duchenne Foundation in 2008 after their first-born son Emilio was diagnosed with Duchenne muscular dystrophy, they could never have imagined how ...

The FDA cited risk of IV infection and kidney toxicity in its letter. The company's CEO said the FDA issued no prior warning for the two concerns.

Duchenne muscular dystrophy (DMD) is the most common muscle disease in children and is passed on by X-linked recessive inheritance. Characteristic is a progressive muscular atrophy. Researchers have ...