NORD has published a new guide to help people and caregivers living with rare diseases understand expanded access and how to access unapproved medicines. In the guide, NORD answers frequently asked questions including …
By Alison Rockett Frase, Guest Blogger In 1995 our son, Joshua, was born with a very rare muscle disorder called Myotubular Myopathy; an early death was imminent for our first-born. …
On July 15, the Food and Drug Administration released the performance goals letter for the reauthorization of the Prescription Drug User Fee Act, also known as PDUFA VI. The PDUFA …
UPDATE: July 11, 2016 Illinois HB 4576 is close to passing! This bill would establish a Rare Disease Commission that would give patients – many of whom are too young …
The following statement was issued by Peter L. Saltonstall, President and CEO of the National Organization for Rare Disorders (NORD), as the Senate announced it would not vote on its …
Time is running out – today may be our last chance before September for anything to happen on the critical Zika package. This Friday, Senators will depart D.C. for their 7-week …
The Commissioner of the U.S. Food and Drug Administration, Robert Califf, M.D., will speak at NORD’s annual Rare Diseases & Orphan Products Breakthrough Summit in October. Commissioner Califf will provide …
Save the date for Sept. 21-22 for a nationwide day of action! Join NORD and 300+ partner organizations as we ‘rally’ for medical research as part of the annual Rally …