Patients & Members – NORD (National Organization for Rare Disorders)

May 6, 2021

TOPIC: Press Releases, Patients & Members, Registries

AAMDSIF and NORD Launch New Natural History Study of Paroxysmal Nocturnal Hemoglobinuria (PNH)

Posted at May 5, 2021 10:00 am by Valaree DonFrancesco

Bethesda, MD, May 6, 2021—The Aplastic Anemia and MDS International Foundation (AAMDSIF) and the National Organization for Rare Disorders (NORD)® today launched the largest-ever study to research Paroxysmal Nocturnal Hemoglobinuria (PNH), a rare bone marrow failure disease . PNH is characterized by the destruction of red blood cells, blood clots and impaired bone marrow function. While there are treatments… Read More

Tags: Registry, Aplastic Anemia and MDS International Foundation, AAMDSIF, Paroxysmal Nocturnal Hemoglobinuria, PNH, Global PNH Patient Registry

April 29, 2021

TOPIC: Featured News, Patients & Members, Advocacy

New Patient Journey Infographic Gives A Glimpse Into The Diagnostic Odyssey

Posted at April 4, 2021 11:33 am by Valaree DonFrancesco

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

Tags: Undiagnosed, diagnostic odyssey, infographic, patient journey

April 8, 2021

TOPIC: Medical, Patients & Members, Industry, Members

NORD Expands Gene Therapy Offerings Through New Online Resources

Posted at April 4, 2021 09:00 am by Lisa Sencen

For several years, the National Organization for Rare Disorders (NORD) has prioritized gene therapy education to advance knowledge and promote safe and effective clinical applications to reduce suffering from rare conditions. With this goal in mind, NORD is dedicated to producing a variety of educational resources for patients, caregivers, health care providers, and the public.

One of the newest resources… Read More

Tags: rare disease community, Gene therapy, NORD, Rare Diseases, continuing medical education, ASGCT

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 2, 2021

TOPIC: Press Releases, Featured News, Patients & Members

NORD and MedicAlert Foundation Team Up to Protect and Empower the Rare Disease Community

Posted at March 3, 2021 10:17 am by Valaree DonFrancesco

Washington, DC — March 2, 2021 More than 25 million Americans living with rare diseases face many challenges, including finding information about their condition, accessing quality health care, and paying for treatments. With help from the National Organization for Rare Disorders (NORDⓇ), the rare community… Read More

Tags: NORD, patient services, National Organization for Rare Disorders, Jill Pollander, MedicAlert, MedicAlert Foundation, Josefina Jervis, MedicAlert Assistance Program

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