Duchenne muscular dystrophy is a genetic disorder characterized by the progressive loss of muscle. It is a multi-systemic condition, affecting many parts of the body, which results in deterioration of the skeletal, heart, and lung muscles.

Duchenne is caused by a change in the dystrophin gene. Without dystrophin, muscles are not able to function or repair themselves properly. Becker muscular dystrophy, which is less severe than Duchenne, occurs when dystrophin is manufactured, but not in the normal form or amount.

Because the dystrophin gene is found on the X-chromosome, it primarily affects males, while females are typically carriers. However, some females can manifest varying ranges of physical symptoms of Duchenne and are therefore called “manifesting carriers”.

PPMD has invested over $55 Million dollars into Duchenne research and in the last two years have contributed to the approval of 8 FDA drug approvals, with many more in development. Contributions to PPMD have a direct impact on the progression of these treatments and bettering the livelihoods for children with Duchenne.