Posted at June 19, 2014 9:12 am by Barbara Hollister
Imagine knowing something is wrong with your baby, but medical professionals cannot provide a diagnosis. Frustrated and scared, you spend every waking moment agonizing about your child’s health and quality of life; you feel helpless and alone. Nobody seems to understand, and some people even insist your child is fine and you are overreacting. Deep down, you know there is something wrong. That is what the Hoffmans experienced after the birth of their son, Ethan. read more >
On behalf of the millions of Americans who have diseases with little or no treatment, the National Organization for Rare Disorders (NORD) has submitted a seven-step plan to the 21st Century Cures Initiative outlining ways to improve the discovery, development, and delivery of medical treatments in the U.S. read more >
June 10th was an emotional – but very educational – day for everyone who attended the Food and Drug Administration’s public meeting on Patient-Focused Drug Development for Inborn Errors of Metabolism. read more >
Posted at June 4, 2014 1:47 pm by Peter L Saltonstall
Biologic therapies have been lifesaving and life changing for the patients we represent – more than 30 million Americans with more than 7,000 rare diseases. Literally, hundreds of thousands of rare disease patients have found their lives bettered by biologics and we are collectively grateful to the innovators and companies that have made this possible. read more >
NORD’s Portraits of Courage Celebration on May 8, 2014, highlighted the many ways in which patients and caregivers display great dignity and grace in living with challenging rare diseases. Patients and families featured in the Portraits of Courage gallery providing an inspiring presence at the event.
We thank our sponsors and supporters who made it all possible, and extend congratulations to our honorees for their accomplishments. NORD will be displaying the Portraits of Courage gallery at other events throughout the year. View our list of sponsors and read the press release listing our honorees.