Entrada Therapeutics has secured authorisation from the UK's Medicines and Healthcare Products Regulatory Agency (MHRA) to ...

In 1987, the muscle protein associated with this gene was named dystrophin. Duchenne muscular dystrophy occurs when that gene fails to make dystrophin. Becker muscular dystrophy occurs when a ...

If you have DMD, the gene that makes a protein called dystrophin is broken. This protein normally keeps muscles strong and protects them from injury. The condition is more common in boys because ...

It is an X-linked recessive disorder that occurs due to changes in the DMD gene, which codes for the protein dystrophin. Dystrophin is a protein that plays a role in strengthening muscle fibers ...

Muscular Dystrophy (MD) is a genetic condition causing progressive muscle weakness and complications in vital organs. Early diagnosis, multidisciplinary care, and emerging treatments like gene therapy ...

The positive results from the second part of the EMBARK trial contrast with findings from one-year post treatment, which did ...

Sarepta's EMBARK study confirms Elevidys offers sustained Duchenne treatment benefits, including motor function improvement ...

The Shanghai-based firm will test the gene therapy candidate in patients between 4 and 9 years old with Duchenne muscular dystrophy.

Roche and Sarepta Therapeutics have shared positive top-line results from a late-stage study of Elevidys (delandistrogene ...

Lead drug PGN-EDO51 targets a mutation in the dystrophin gene that can be treated by skipping exon 51, making it a potential rival to Sarepta Pharma's Exondys 51 (eteplirsen), which made $454 ...

Roche has reported positive results from the second year of the EMBARK Phase III trial of Elevidys (delandistrogene moxeparvovec), the first approv ...