Featured News Archives - NORD (National Organization for Rare Disorders)

April 7, 2021

TOPIC: Featured News, Patients & Members

Ray Stevens is Taking Rare Disease Awareness to New Heights

Posted at April 4, 2021 09:25 am by Valaree DonFrancesco

In 2001, Ray was inspired by a family he met whose daughter was diagnosed with phenylketonuria (PKU). He translated his academic knowledge into clinical treatments and was able to help create two new medicines for this devastating rare disease. Then in 2007, his daughter, who was four years old at the time, was diagnosed with Kawasaki disease, a rare… Read More

Tags: Undiagnosed, awareness, Mount Everest, Ray Stevens, Kawasaki disease, Undiagnosed Day, World Health Day

March 25, 2021

TOPIC: Press Releases, Featured News, Research, Industry

New Study Investigates the Number of Available Orphan Products, Generics and Biosimilars

Posted at March 3, 2021 09:29 am by Jennifer Huron

Washington, DC, March 25, 2021—The National Organization for Rare Disorders (NORD®) today announced the findings of a new study that details the number of orphan products, generics and biosimilars available to treat rare diseases. NORD commissioned Avalere to conduct the analysis to examine if laws and regulations are helping to bring… Read More

Tags: FDA, Orphan Drug Act, orphan drugs, drug development, Patent, Orphan products, biosimilars, RareInsights, US Food and Drug Administration, Avalere, generics

March 23, 2021

TOPIC: Featured News

Save On Quality, Vetted PPE with NORD and Project N95

Posted at March 3, 2021 06:00 am by Valaree DonFrancesco

To help rare disease community members who are having trouble acquiring quality, vetted PPE, the National Organization for Rare Disorders (NORD)® is collaborating with the nonprofit Project N95 so you can purchase N95 respirators (certified by CDC and NIOSH), nitrile powder free gloves, Level 1 AAMI isolation gowns, ASTM Level 1 and Level 3 surgical… Read More

Tags: NORD, covid-19, PPE, project n95

March 16, 2021

TOPIC: Press Releases, Featured News

NORD Joins with the Krabbe Disease Community to Publish “Voice of the Patient” Report

Posted at March 3, 2021 02:18 pm by Valaree DonFrancesco

Washington, DC, March 16, 2021—The National Organization for Rare Disorders (NORD)®, together with KrabbeConnect, The Legacy of Angels Foundation, Partners for Krabbe Research, Hunter’s Hope and with additional support from Gain Therapeutics, Magneta Therapeutics, PassageBio and Neurogene, has published the “Voice of the Patient” report sharing the stories of patients and families impacted by Krabbe disease, also known as… Read More

Tags: press release, Krabbe disease, Voice of the Patient, externally-led Patient-Focused Drug Development Meeting, EL-PFDD, krabbe

March 4, 2021