Published August 18, 2014 by NORD
The day Joshua Frase was born, his parents Alison and Paul, were given grave news about their newborn’s life – he most likely would not survive that day. His weakened …
Continue reading “The Will to Live and the Strength for a Cure”
Read morePublished June 17, 2014 by NORD
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 …
Continue reading “NORD Recommendations for Advancing Drug Discovery, Development, and Delivery”
Read morePublished June 4, 2014 by NORD
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 …
Continue reading “Thoughtful Biosimilars Policy Is Best Way to Ensure Optimum Patient Outcomes”
Read morePublished May 30, 2014 by NORD
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 …
Continue reading “Images from the Portraits of Courage Celebration”
Read morePublished April 21, 2014 by NORD
3-D printing is an exciting technology that has shown great promise in the treatment of several rare disorders. Bioprinting is a form of 3-D printing in which cells are printed …
Continue reading “Engineering Treatments for Rare Disorders”
Read morePublished February 28, 2014 by NORD
The Food and Drug Administration’s Office of Orphan Products Development (OOPD), in collaboration with the FDA Center for Drug Evaluation and Research (CDER), has launched a web-based educational tool for …
Continue reading “FDA Creates New Web-Based Educational Tool About the Agency”
Read morePublished December 16, 2013 by NORD
CAMBRIDGE, MA and DANBURY, CT—-Genzyme, a Sanofi company, and the National Organization for Rare Disorders (NORD) today announced the creation of a fund to pay for standard diagnostic testing for …
Read morePublished December 4, 2013 by NORD
Genome editing is a process that involves cutting out mutations in DNA and replacing them with new genetic material. This is an exciting idea because if it can be refined …
Continue reading “A CRISPR Approach to Genome Editing”
Read morePublished October 23, 2013 by NORD
NORD recently posted the following question on its Facebook page: How long did it take you or a loved one to get an accurate diagnosis? We were stunned at …
Read morePublished September 4, 2013 by NORD
Even though Gaucher disease is one of the better known rare diseases and diagnostic testing is available, some patients still go for 10 years or longer without getting an accurate …
Continue reading “Even “Well Known” Rare Diseases Pose Diagnosis Problems”
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