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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

April 22, 2021

TOPIC: Press Releases, Medical, Research

CDISC and NORD Partner to Develop Data Standards for Rare Diseases

Posted at April 4, 2021 11:30 am by Valaree DonFrancesco

Danbury, CT and Austin, TX – April 22, 2021 – CDISC and the National Organization for Rare Disorders (NORD®) have announced a partnership to develop global data standards for rare diseases. The data standards will be released in a Therapeutic Area User Guide that will be available at no cost on the CDISC website for researchers to leverage in… Read More

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

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

March 4, 2021

TOPIC: Press Releases, Featured News, Research, Industry

New Report Finds Medical Treatments for Rare Diseases Account for Only 11% of US Drug Spending; Nearly 80% of Orphan Products Treat Rare Diseases Exclusively

Posted at March 3, 2021 11:35 am by Jennifer Huron

Washington, DC, March 4, 2021—As US policymakers consider options to address rising health care costs while still meeting the needs of patients, a new report from IQVIA has found that rare diseases account for just 11% of medical invoice spending in the United States, and 79% of all orphan products treat only rare diseases. The report also highlights… Read More