Researchers at the National Institutes of Health and their colleagues have found that a toxic protein made by the body called DUX4 may be the cause of two very different rare genetic disorders. For ...
A milestone in the treatment of Duchenne muscular dystrophy (DMD) passed recently with remarkably little public notice. That ...
Muscular dystrophy, known as DMD, is a rare and fatal genetic disease that primarily affects boys, slowly robbing them of their ability to walk, breathe and live independently. Until now, there have ...
Disability advocate TaLisha Gryzb is a mother of four living with muscular dystrophy who educates viewers about her experiences on her Youtube channel.
The FDA granted accelerated approval to delandistrogene moxeparvovec (Elevidys), the first gene therapy for Duchenne muscular dystrophy, the agency announced Thursday. The adeno-associated virus-based ...