Written by Valaree DonFrancesco on June 26, 2020
NORD is deeply concerned about the “Promising Pathway Act” recently introduced in both the Senate and the House. Contrary to the stated goal of giving patients with serious and life-threatening diseases a “chance at receiving meaningful treatments,” this legislation, by its own terms, would lower FDA’s approval standards, exposing patients… Read More
Written by Valaree DonFrancesco on March 30, 2020
The National Organization for Rare Disorders (NORD®) and EURORDIS-Rare Diseases Europe share the global concern that diagnostics, therapeutics and vaccines to address COVID-19 be developed expeditiously. NORD and EURORDIS represent the 30 million people in the US and 30 million people in the EU living with rare diseases, individuals disproportionately… Read More
Written by Valaree DonFrancesco on February 3, 2020
An essay by NORD President and CEO Peter L. Saltonstall on the Orphan Drug Act is featured in Congressional Quarterly Researcher’s Conquering Rare Diseases special issue.
“Rare disease patients cannot afford to return to the early 1980s. The more than 25 million rare disease patients in the United States are counting… Read More
Written by Valaree DonFrancesco on December 2, 2019
Do you have questions about gene editing and how it may advance the treatment of people with rare diseases? Much has been written lately about the promise of CRISPR and other gene-editing tools as future treatments for many rare diseases.
NORD is creating a new educational video… Read More
Written by Valaree DonFrancesco on October 29, 2019
On the heels of the 2019 Rare Diseases and Orphan Products Breakthrough Summit, NORD’s Rare Action Network (RAN) Volunteer State Ambassadors made their way to Capitol Hill on October 23, 2019, to tell their stories and show their stripes. Ambassadors from 20 states… Read More