In a new study published in Science, a Belgian research team explores how genetic switches controlling gene activity define ...

According to the World Health Organization (WHO), hundreds of millions of new cases of bacterial sexually transmitted ...

Biomedical engineers at Duke University have demonstrated a promising new approach that could be used to treat a rare and ...

No doubt 2025 will usher in many more advances in CRISPR therapies, thanks to the FDA’s new Platform Technology Designation ...

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 ...

The phase 3 EMBARK trial has been evaluating the gene therapy in ambulatory boys aged four to seven years with a confirmed mutation in the DMD gene. Estimated to affect one in every 5,000 male births ...

Elevidys, which is the only gene therapy for Duchenne muscular dystrophy on the market, delivers a gene that encodes microdystrophin, an engineered protein developed by Sarepta that it says can carry ...

Also Read: Solid Biosciences Outpaces Competitors In Duchenne Gene Therapy Development Despite being one year older (average age 7.18 years) than those treated in Part 1 (average age 5.98 years ...

An inherited disorder affecting primarily boys, DMD is characterized by the progressive loss of skeletal, heart and lung muscle. It is caused by a mutation in the gene for the dystrophin protein, ...

Gene therapies have set a high bar for outcomes in pharma by delivering curative treatments for difficult diseases in a one-and-done punch. But for Duchenne muscular dystrophy, the results of ...

While DMD is currently incurable, hope lies in emerging treatments like ELEVIDYS (Delandistrogene Moxeparvovec Rokl), a gene therapy designed to help the muscles produce dystrophin. However ...