Gene therapy has emerged as a promising approach to previously untreatable conditions, including Duchenne muscular dystrophy (DMD), a progressive disorder caused by DMD mutations that leads to early ...

A new study led by researchers at Hospital for Special Surgery (HSS) suggests that currently available therapies may help ...

In 2021, 529 million people were living with diabetes worldwide, and an estimated 1·31 billion individuals are projected to ...

The CHMP recommends conditional marketing authorization for Duvyzat to treat Duchenne muscular dystrophy in patients aged 6 ...

A new study led by researchers at the Hospital for Special Surgery (HSS) suggests that currently available therapies may help ...

Italfarmaco's oral HDAC inhibitor Duvyzat has been recommended for approval in the EU as a treatment for Duchenne muscular ...

EMA grants conditional marketing authorization to Italfarmaco’s Duvyzat to treat Duchenne muscular dystrophy: Amsterdam, The Netherlands Monday, April 28, 2025, 16:00 Hrs [IST] ...

The EC has granted orphan drug designation to Dyne Therapeutics’ DYNE-251 for treating Duchenne muscular dystrophy (DMD).

It is designed to enable targeted muscle tissue delivery and promote exon skipping in the nucleus ... by the U.S. Food and Drug Administration for the treatment of DMD mutations amenable to exon 51 ...

It's been nearly two years since Elevidys, a gene therapy for Duchenne MD developed by Sarepta Therapeutics, was approved by ...