Urge the FDA to Say YES to Accelerated Approval For Therapies For Duchenne.

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We urge the Food and Drug Administration (FDA) to use the Accelerated Approval pathway for approval and access to safe, effective therapies for Duchenne Muscular Dystrophy – the leading genetic killer of children that impacts 1 out of every 3,500 boys born in the United […]

Meeting: Characterizing and Communicating Uncertainty in the Assessment of Benefits and Risks of Pharmaceutical Products

INstitute of Medicine

When: February 12, 2014 – February 13, 2014 (9:00 AM Eastern) Where: Food and Drug Administration (FDA) Campus (Building 31, Great Room ) • 10903 New Hampshire Avenue, Silver Spring, MD 20993 Topic: Biomedical and Health Research Activity: Forum on Drug Discovery, Development, and Translation […]

Keck Medicine Physicians First to Implant Epilepsy-Controlling Device


Keck Medicine of USC has become the world’s first medical center to implant a responsive brain device newly approved by the Food and Drug Administration (FDA) to treat epilepsy. It has the potential to help millions of people worldwide. The treatment involves surgical implantation of […]

FDA Sets Up New Rare Disease Research Fund


The US Food and Drug Administration (FDA) is to support the research of treatment for rare diseases through a new $14m fund. The money will support 15 research projects that are investigating new ways to tackle rare diseases, defined in the US as conditions that affect […]

FDA Reform Can Lift U.S. Economy


By Tomas J. Philipson and Andrew von Eschenbach. Without a growing economy that creates more high-paying jobs, both President Barack Obama and congressional Republicans face the unpalatable prospect of much higher taxes or reductions to popular programs (or both). Growth is the best way to cut […]

CureTheProcess Seeks Survey Participation for Phase II: Deadline October 26

The CURETHEPROCESS Campaign strives to inspire science-driven public policy that will increase the predictability of the regulatory process for rare disease treatments.

Complete the Survey & win a $50 Gift Card! Over the last three years, more than 180 patient organizations, physician societies and medical institutions have partnered with us to improve the FDA’s regulatory process for rare diseases through our CureTheProcess Campaign. We have been very […]

FDA To Hold Series of Public Meetings To Pick 20 Diseases To Focus on Over Next Five Years; Few Rare Diseases On Preliminary List

The public is being invited to share their input to the FDA on October 25, 2012.

On October 25, 2012, the FDA will provide an overview of Patient-Focused Drug Development and the public is invited to comment and share their voice. This will be the first of many public meetings held by the FDA over the next five years with a focus […]