Duchenne muscular dystrophy (DMD) is a severe genetic condition characterized by progressive muscle weakening. People with DMD typically only live into their 20s, but life expectancy has been ...

Cardiomyopathy is one of the main causes of death for people with DMD. What Causes It? An abnormality in the DMD gene causes Duchenne muscular dystrophy. The DMD gene produces dystrophin ...

While self-care is different for everyone, studies show that people who adapt well to ... “Quantifying the burden of caregiving in Duchenne muscular dystrophy.” Muscle & Nerve: “Burden ...

Progress in health and social care in European countries is helping many boys with Duchenne muscular dystrophy (DMD ... views on how to improve lives of people with the condition across Europe.

Deb Jenssen has two girls showing signs of Duchenne muscular dystrophy, a disease that for so long, people thought only affects boys. Shut out of clinical trials, the family struggles to find ...

including the Muscular Dystrophy Association, which funds research and supports people with neuromuscular diseases, are lobbying states nationwide to add a genetic screen for Duchenne's to their ...

As the name suggests, Duchenne muscular dystrophy is caused by a defective gene for dystrophin (a protein in muscles). However, it often occurs in people with no known family history of the condition.

There is a simple discount patient access scheme for vamorolone. NHS organisations can get details on the Commercial Access and Pricing (CAP) Portal. Non-NHS organisations can contact ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that ... of gene therapy for Duchenne muscular dystrophy, raising more ...

Yet a major benefit is that the gene therapy provides another option for people with Duchenne muscular dystrophy, and it’s administered just once. There is no cure for Duchenne muscular ...

Some types of muscular dystrophy cause irregular heartbeats. Gene therapy. Delandistrogene moxeparvovec (Elevidys) is a gene therapy approved to treat Duchenne MD. It's only prescribed to people ...

Vamorolone (Agamree) is available on the NHS as a possible treatment for Duchenne muscular dystrophy (DMD) in people 4 years and over. Is this treatment right for me? Your healthcare professionals ...