—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 ...

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

WASHINGTON, April 23, 2024 /PRNewswire/ -- Parent Project Muscular Dystrophy (PPMD), a US nonprofit organization, and the UK charity Duchenne UK, two leading organizations dedicated to ending Duchenne ...

Duchenne muscular dystrophy has several approved drugs, including a gene therapy that provides children who have the rare, inherited muscle-wasting disease the option of a one-time treatment. But each ...

HHS has announced the addition of Duchenne muscular dystrophy and metachromatic leukodystrophy to the Recommended Uniform ...

A diagnosis of Duchenne muscular dystrophy (DMD) can come as a shock to parents and caregivers. Resources and support from family and friends can help you and your child navigate the diagnosis and ...

Interim data showed 110% average microdystrophin expression and up to 88% dystrophin-positive muscle fibers in the first three participants. Two patients saw an 8% average heart function improvement ...

Solid Biosciences Inc. (NASDAQ:SLDB) is one of the stocks that will double in 2026. On December 4, Needham analyst Gil Blum initiated coverage of Solid Biosciences with a Buy rating and $16 price ...

Tracing the impact of a single protein, Piezo1, researchers found that restoring it in muscles affected by Duchenne muscular dystrophy could improve their ability to heal efficiently. One protein, ...

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 ...

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 ...