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Archives for: November 19th, 2018

Leveraging Real-World Treatment Experience from Expanded Access Protocols

Written by Christina Jensen on November 19, 2018

Building on the Reagan-Udall Foundation for the FDA’s work with FDA and other stakeholders to develop the Expanded Access Navigator, the Reagan-Udall Foundation is convening stakeholders from government, industry, academia and patient groups to discuss process issues that include:

  • Increase understanding of the expanded access program
    • Identify and discuss strengths, challenges… Read More

FDA Announces First Gene Therapy in U.S.

Written by Mary Dunkle on August 31, 2017

In an action the rare disease community has been eagerly awaiting, the Food and Drug Administration (FDA) has announced the first-ever approval of a gene therapy in the U.S. The product is Kymriah, and it is approved to treat certain children and young adults who have a cancer of blood… Read More

Advance the Dialogue at the NORD Rare Summit

Written by Christina Jensen on August 15, 2017

It has been an interesting year for healthcare reform and the Orphan Drug Act, and as I’m sure you are aware; the conversation isn’t over yet. Major changes to healthcare and tax reform continue to be discussed and that is why it is so important that now, more than ever, rare… Read More