Galactosemia is a rare, hereditary disorder that affects the body’s ability to convert galactose to glucose. Galactosemia affects 1 in every 40,000 to… Read More
The National Organization for Rare Disorders (NORD®) is pleased to support California Attorney General Xavier Becerra’s nomination as the next Secretary of the Department of Health and Human Services (HHS). Rare disease patients rely on close collaboration between HHS and numerous agencies under its purview, including the Food and… Read More
By Debbie Drell, Director of Membership
My sister was diagnosed with a rare disease, pulmonary hypertension, in September of 1998. At some point during her diagnosis, she was told she wouldn’t live to see her 30th birthday. She was 28. There was one… Read More
Read at Pyruvate Kinase Deficiency (PKD) Externally-Led Patient Focused Drug Development Meeting (PFDD) Meeting on Friday, September 20.
Written By Debbie Drell, Director of Membership, NORD
According to the NIH, more than 500 affected families have been identified with Pyruvate Kinase Deficiency (PK Deficiency) in the world, but just 140 miles north… Read More
NORD and the Foundation for Rare Blood Diseases (SZB) invite you to register for an externally-led Patient Focused Drug Development (EL-PFDD) meeting on Pyruvate Kinase deficiency (PKD), taking place Friday, September 20. Scholarships are available to… Read More
