TOPIC: Featured News, Patients & Members, Advocacy
The road to a correct rare disease diagnosis is far too long. This Undiagnosed Day, please share this infographic with your community and if you, or someone you know, is in need of information or resources to assist you on your diagnosis journey, please visit our Undiagnosed Rare Disease… Read More
TOPIC: Featured News, Patients & Members
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
TOPIC: Press Releases, Featured News, Research, Industry
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
TOPIC: Featured News
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
TOPIC: Press Releases, Featured News
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
