Rare Disease Report: event highlights from World Orphan Drug Congress USA

Rare Disease Report logo x150Rare Disease Report was a media partner of this year's World Orphan Drug Congress USA. They posted some interesting articles as a result of attending the show. These are posted below, with thanks to Rare Disease Report.

Sarepta Wants to Change the Way We Do Business with the FDA

At the World Orphan Drug Congress USA, CEO Chris Garabedian of Sarepta Therapeutics gave the audience want they wanted – a bold talk about shaking up the way the pharmaceutical industry works with the FDA.  And the audience loved it… Read more here.

It Takes a Village.  Developing Orphan Drugs with Bristol-Myers Squibb.

The company Bristol Myers Squibb (BMS) is not usually associated with orphan drugs. Surprisingly, they have been instrumental in several orphan drug developments and those drugs, and the stories behind those drugs, were discussed by Simeon Taylor, PhD, MD, Vice President of Research and Development, Scientific Affairs at BMS at the World Orphan Drug Congress USA…. Read more here.

John Crowley Talks PDUFA V – Will it Do What it Was Designed to Do?

On the last day of the World Orphan Drug Congress, John Crowley, Chairman and CEO, Amicus Therapeutics provided an inspiring overview of how the Fifth Prescription Drug User Fee Act  (PDUFA V) and new government policies may affect the orphan drug industry… Read more here.

For more information on World Orphan Drug Congress USA 2014, download the prospectus now.