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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 Speakers Bureau

Written by Mary Dunkle on June 14, 2016

Need a Speaker for Your Classroom or Event? Contact NORD!


Teachers, medical school professors, and community members seeking speakers to talk about rare diseases in the classroom or at community events can contact NORD for help. Working closely with its 250 member organizations, NORD now has a Patient/Caregiver Speakers Bureau to… 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

NORD Establishes Rare Disease Patient/Caregiver Speakers Bureau

Written by Mary Dunkle on January 18, 2016

speakers-bureau-quoteThe National Organization for Rare Disorders (NORD) has established a Patient/Caregiver Speakers Bureau through which volunteers will share their stories to promote better understanding of the challenges of living with a rare disease.

Speakers will be paired with opportunities to speak in… 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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