Duchenne Muscular Dystrophy (DMD) is a genetic disease in boys, which has a devastating impact on both patients and their families. Despite advances in medical management and therapeutic development, disease progression is relentless and boys typically lose the ability to walk in their teens and suffer from increasing degree of cardiac and respiratory failure by the third decade of life. DMD occurs due to gene mutations resulting in the loss of the Dystrophin protein. A functionally near identical backup copy of the protein called Utrophin or Dystrophin-Related Protein exists, however, it is expressed at low levels that are insufficient to prevent disease formation. Recently FDA-approved DMD treatments are mutation-specific and are applicable to only Approx. 20% patients. Currently there is no cure for DMD. Thus, there is a great unmet clinical need for therapies effective in all patients of DMD.
We at MyoArete are committed toward developing therapies for treating all DMD patients. We have developed platforms to identify and develop utrophin upregulation-based treatments for DMD. These treatments are predicted to improve function, slow disease progression and increase lifespan in all DMD patients, regardless of mutation status.
DMD by the Numbers
DMD is estimated to occur in 1: 3500 boys born worldwide.
2/3 cases have a family history while 1/3 cases are sporadic.
17,000 DMD patients are estimated to currently live in the US and Approx. 50,000 DMD patients live in the US and Europe combined.