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
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
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.
Written by Mary Dunkle on December 2, 2015
Education has always been a key component of NORD’s mission and this year we expanded our outreach to two vitally important audiences – medical professionals and students… Read More
Written by Mary Dunkle on September 2, 2015
A few months ago, NORD and our members hosted a “Meet the Patients” event at the annual conference of the American Medical Student Association (AMSA). We were thrilled with the number of students who came to our booth and the interest they displayed.
Since then, more than 100 students have signed… Read More