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:

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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…