Dear Friends,

This community should be proud of the role we have played over the last several weeks preparing for the potential approval of multiple therapies to treat Duchenne. As I mentioned last week, there is still…

Dear Friends,

It has been an incredibly intense few months as we hit the homestretch of 2015 and head into a promising 2016. One Advisory Committee meeting is down and we have another quickly approaching most likely in late January 2016.…

I have never been so proud of this community. Today our community reached a historic milestone, a moment I wondered if we would ever see. Today’s FDA Advisory Committee (Ad Comm) for BioMarin’s drisapersen, gave us an up close look at the regulatory process, learning firsthand what the…

Tomorrow, November 24, 2015, is a historic day for the Duchenne community as we enter into our first Advisory Committee Meeting with the FDA. While some of you may be attending in person or even participating, we know that many of you are curious as to how the process will unfold throughout the day. 

Watch a…

Dr. Stu Peltz from PTC Therapeutics shares his gratitude to the Duchenne community for the contribution families have made to the development of their therapy, Translarna.

Read Dr. Peltz's letter:

You Are…

For those of us who’ve been in this community for decades, at some point we began to believe that we’d never see the day when products that had been designed specifically with the intent to treat individuals with Duchenne would be reviewed by the FDA. But here we are—standing on the shoulders of the…

By now, you probably know what to expect from the upcoming Advisory Committee meetings in terms of logistics: how to submit written testimony, how to apply for an open public hearing slot, order of the day, etc. (read what an Ad Comm is or …

For over a year, PPMD has been working with congressional champions and industry partners to help lead an effort to streamlining the regulatory pathways and review processes for targeted therapeutics (which would include such things as 'follow on exons') by clarifying the FDA’s existing authority to leverage data previously used in the approval of a targeted…

We have a running joke in our family that Alex and I argue like an old married couple. No offense intended to those who can identify, but we do, and more often than I care to admit. Sometimes our discussions (as we call them) are silly, and sometimes they have merit. Sometimes the exchanges are brief, and sometimes a bit extended. But, what do you expect when two…

Many of you have seen – and signed – a petition that circulated recently about preventing Medicare from applying competitive bidding prices to complex technology; an action that will likely reduce access to wheelchair features many of our family members depend on. This effort has now evolved into a legislative effort and we urge all members of our…