Clinical trials are research projects, performed as a way of bringing about progress in medicine and research. Clinical research projects make it possible to scientifically assess the properties of a study medication, to demonstrate the effectiveness and safety of a treatment or a medical device, or to learn more about a disease and methods to diagnose and monitor it.

Here at MAC we are looking for people who would like to take part in our future clinical trials researching potential new treatments for Duchenne muscular dystrophy.

Duchenne muscular dystrophy (DMD), also known as Duchenne’s, DMD disease, or Duchenne disease, is one of the most common muscular dystrophy types. It is a genetic (inherited) condition, mainly affecting males with Duchenne muscular dystrophy life expectancy being in their 30’s, the charity Duchenne UK estimates there are 2,500 people living with the condition in Britain.

Duchenne muscular dystrophy causes the body to make a much shorter, inactive version of a protein known as dystrophin, which is essential for protecting the working muscles and results in progressive muscle weakness, other Duchenne muscular dystrophy symptoms often include scoliosis (curving of the spine), dilated cardiomyopathy and respiratory problems. Physiotherapy, exercise, and medications, such as corticosteroids, are the most usual Duchenne muscular dystrophy treatments, but there is currently no cure for the condition.

There is an urgent need for additional treatments to help those living with the condition. Current research on Duchenne muscular dystrophy treatments often focuses around DMD clinical trial gene therapy or exon skipping therapy.

More information will be available on the clinical trial once approval of the research is granted by the regulatory bodies in the UK.