Michelle C. Werner agonized over whether the gene therapy treatment Elevidys was right for her teenage son. But to him, the ...

A possible gene therapy approval and a series of major trials readouts set the stage for a busy year of drug development in Duchenne muscular dystrophy.

It causes less severe problems than the most common type, Duchenne muscular dystrophy. Becker MD symptoms can range ... or maintain a running pace have trouble lifting heavy loads have calf muscles ...

Some people live a full life with MD. Others, including those diagnosed with Duchenne muscular dystrophy (DMD), typically ...

Prenatal diagnostic testing can now determine whether a fetus carries a debilitating or fatal sex-linked mutation. But with such screening, why hasn't the disease allele frequency gone down ...

Pfizer is “evaluating appropriate next steps” for its Duchenne muscular dystrophy (DMD) gene therapy after it failed a phase 3 trial. Fordadistrogene movaparvovec (PF-06939926) was unable to ...

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 ...

Hudson Sanford is a seven-year-old boy that was diagnosed with Duchenne muscular dystrophy (DMD) when he was four years old.

July 24, 2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores full-length dystrophin protein, which could lead to new treatments for ...

WASHINGTON, Aug. 27, 2024 /PRNewswire/ -- Marking a significant milestone in improving the lives of children affected by Duchenne muscular dystrophy (Duchenne), Parent Project Muscular Dystrophy ...

Italfarmaco's marketing application for HDAC inhibitor givinostat as a treatment for Duchenne muscular dystrophy (DMD) has been accepted by the EMA, setting up an approval decision in 2024.

KUWAIT, Sept 11: Dr. Laila Bastaki, Head of the Genetic Diseases Center at the Ministry of Health, emphasized the importance of incorporating genetic testing for Duchenne muscular dystrophy (DMD) into ...