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Archives for: August 31st, 2017

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

NORD Members Share Great Resources for ER Visits During #RareERChat

Written by Mary Dunkle on May 27, 2016

Emergency room visits are a fact of life for many people with rare diseases. And, when the patient is a child, these experiences can be especially stressful for the entire family.

For that reason, NORD was delighted to host a tweetchat with Texas Children’s Hospital –… Read More

FDA Invites Patient Organizations to Organize Their Own Meetings on the PFDD Model

Written by Mary Dunkle on January 12, 2016

When the Patient-Focused Drug Development (PFDD) Initiative was first announced as part of the 2012 PDUFA reauthorization, many patient organizations expressed concern that the initiative’s public meetings would focus on just 20 groups of diseases. However, FDA has now provided a pathway for extending the model to many other diseases.

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