The National Institutes of Health (NIH) is one of the biggest funders of medical research in the US, and projects it has funded have touched almost every Duchenne therapeutic in development now in some way. In FY2016, the NIH is investing nearly $32.3 billion in medical research, and of…
ContinueAdded by Abby Bronson on March 24, 2016 at 9:30am — No Comments
At long last we have received information regarding the rescheduled Advisory Committee for Sarepta (eteplirsen). The Ad Comm will take place on Monday, April 25 at the College Park Marriott Hotel & Conference Center (a non-FDA campus…
ContinueAdded by PPMD on March 10, 2016 at 9:00am — No Comments
Added by PPMD on March 10, 2016 at 9:00am — No Comments
Gene therapy.
Those two words have been tossed around for some time, starting in the late 1990s when several gene therapy trials started, but ended with fatal results. Now, when the field has learned a lot from those initial results and with optimized technologies, we come back to gene therapy as a viable option for genetic diseases. This renewed…
ContinueAdded by Abby Bronson on March 8, 2016 at 12:00pm — 4 Comments
For 22 years, PPMD has brought together families with leading experts, care providers, and industry partners through our Annual Connect Conference, FACES…
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