The Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) is an FDA-funded initiative with the goal of accelerating therapy development across rare diseases.
At the age of 14, I was diagnosed with a rare form of GI tract cancer called gastrointestinal stromal tumors (GIST). Living with a rare disease is incredibly complicated, and you must learn how to be your own …
I have been a healthy person my whole life. I never drank alcohol or smoked, and I had a good diet. In 2004, I began to have some questionable numbers on blood work, but my primary doctor could never really tell what was going on. My …
Every September, we celebrate and raise awareness for newborn screening (NBS) in the United States and the impact it has had on millions of families. Each year, approximately four million babies are screened for …
TUCSON, Ariz. and WASHINGTON, September 1, 2021 — For two years, Critical Path Institute (C-Path), the National Organization for Rare Disorders (NORD) and the U.S. Food and Drug Administration (FDA) have joined …
August 27, 2021 – Registration is now open for the NORD Rare Diseases and Orphan Products Breakthrough Summit®. The NORD Summit, one of the largest global events in rare disease, brings together experts …
In 2001, I was diagnosed with ocular melanoma. What makes ocular melanoma rare is that only 2,500 people are diagnosed with it each year. Following my diagnosis in October of 2001, I went through numerous tests at Sylvester Cancer Center. In November, I was admitted into the hospital where …
Research study is open to participants worldwide to advance understanding and treatments for Metachromatic Leukodystrophy, a recessive genetic disorder that is characterized by the accumulation of fats (lipids) called sulfatides …
Last month, the National Organization for Rare Disorders (NORD) hosted their second virtual Living Rare, Living Stronger NORD Patient & Family Forum. From June 25-28, enthusiastic attendees from around the …
Washington, DC – The National Organization for Rare Disorders® (NORD) was named an Official Charity Partner for the 2021 TCS New York City Marathon, taking place on November 7. This year serves …