Ryan Fischer's Blog – May 2014 Archive (3)

PPMD Congressional Champions Voice the Need for Action on the MD-CARE Act

On May 20th, the House Energy and Commerce Subcommittee on Health held a hearing entitled, 21st Century Cures: The President’s Council of Advisors on Science and Technology (PCAST) Report on Drug Innovation.

The hearing was led by Chairman Joe Pitts (R-PA) and is part of the new 21st Century Cures initiative that seeks to accelerate the discovery, development and delivery of promising new treatments to patients.…

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Added by Ryan Fischer on May 22, 2014 at 3:30pm — No Comments

Unprecedented Collection of Duchenne Experts Developing Guidance for FDA

The process for writing a Draft Guidance on Duchenne for the FDA and industry is in full swing.

As you know, the purpose of this guidance is to assist…

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Added by Ryan Fischer on May 9, 2014 at 9:30am — No Comments

MD-CARE Act Results: Exon Skipping from Lab to Patient

Originally signed into law in 2001, the MD-CARE Act has birthed key discoveries in the muscular dystrophies, including the scientific justification for why exon skipping therapies could potentially work in humans.

Without it being…

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Added by Ryan Fischer on May 8, 2014 at 4:30pm — No Comments

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