Researchers at Baylor College of Medicine, the Jan and Dan Duncan Neurological Research Institute (Duncan NRI) at Texas ...

A team led by University of Pittsburgh School of Public Health geneticists has shown, for the first time, that a gene "silencer" that resides in junk DNA is directly sparing people from a devastating ...

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 ...

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, ...

The company, which secured $200 million from investors in a private funding deal last week, learned Tuesday of a serious side effect experienced by one participant on a high dose of its Rett therapy.