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 ...

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

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 ...

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.

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

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 ...

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 ...

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 ...

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

KUWAIT CITY, Sept 11: Assistant Undersecretary for Medical Support Services at the Ministry of Health, Dr. Abdullah Al-Fars, announced on Wednesday that Kuwait is the first country in the Middle East ...

Patients treated with 20 mg/kg of DYNE-251 Q4W had a mean absolute dystrophin expression of 3.71% of normal. Dyne is initiating registrational cohorts in the DELIVER trial and plans to provide an ...