PPMD's Blog – November 2015 Archive (4)

November 24: FDA Advisory Committee Meeting for Drisapersen - Live Webcast Information

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…


Added by PPMD on November 23, 2015 at 2:00pm — No Comments

PTC Thanks the Duchenne Community

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…


Added by PPMD on November 18, 2015 at 3:00pm — No Comments

FDA Advisory Committee Scenarios

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 …


Added by PPMD on November 17, 2015 at 1:00pm — No Comments

PPMD Supports Advancing Targeted Therapies for Rare Diseases Act of 2015 (S. 2030)

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…


Added by PPMD on November 12, 2015 at 1:09pm — 1 Comment

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