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Featured Blog Posts – November 2015 Archive (3)

Staff A Historic Day for Duchenne

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…

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Added by Pat Furlong on November 24, 2015 at 8:30pm — 2 Comments

Staff 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 …

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

Staff 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…

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