Duchenne is getting a great deal of attention these days. With the excitement around the Sarepta trial and more trials coming online, more attention to Duchenne is a great thing as we continue to raise awareness. Personal stories about families only help to educate the public on Duchenne. It allows them to relate and perhaps to think, If this was my son, what would I do??

A recent…

PPMD Taking Action to Avert Sequestration

Those engaged in federal advocacy probably thought the Prescription Drug User Fee Act and its acronym PDUFA would take the cake in terms of policy jargon. But as the nation's eyes are fixed on…

PPMD and other groups submit joint statement making the case for neuromuscular disorders affecting children

As many advocates know from earlier blog postings, PPMD has been deeply and passionately engaged to enhance how the Food and Drug Administration (FDA) goes about its business of reviewing potential therapies and treatments for patients with Duchenne. Our work is focusing on ensuring that the agency moves as expeditiously as possible and…

Today, Parent Advocate and Founder of Two Smiles One Hope Foundation, Alison Willis and I represented the Duchenne community at the Congressional Rare Disease Caucus Briefing in Washington, DC. Alison did a remarkable job speaking on behalf of PPMD regarding the Patient Participation in Medical Product Discussions Provision within the newly passed…

Tomorrow, we will head to the polls to choose who will lead our nation for the next four years.

While many of us – particularly those of you who live in key "swing" states that will likely determine the outcome of the election – may be tired of the unceasing television commercials, robocalls and mailers, it's important to recognize that regardless of who is elected, creating…