Summit Therapeutics announced positive interim data from a Phase 1 clinical trial evaluating a new oral formulation of its lead utrophin modulator, SMT C1100, for Duchenne. In this dose escalating clinical trial's…

PPMD is thrilled to see the continued progress of VBP-15, now known as Vamorolone. Progress to date includes a first in-human study of healthy volunteers and animal data which suggests it has none of the side effects of steroids, does not impact linear growth, and does not promote…

The Duchenne community is working closely together to prepare for the April 25 Advisory Committee Meeting for Sarepta’s therapy eteplirsen. PPMD, the Duchenne Alliance, the Center for DMD at UCLA, Jett Foundation, and the Race to Yes…

At long last we have received information regarding the rescheduled Advisory Committee for Sarepta (eteplirsen). The Ad Comm will take place on Monday, April 25 at the College Park Marriott Hotel & Conference Center (a non-FDA…

UPCOMING AD COMMS

Sarepta…

For 22 years, PPMD has brought together families with leading experts, care providers, and industry partners through our Annual Connect Conference, FACES…

Parent Project Muscular Dystrophy's Annual Connect Conference is a unique convergence of industry partners, scientific leaders, medical providers, people living with Duchenne, and their families. This exceptional meeting has grown to…