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March 22, 2021

TOPIC: Research, Registries

The Cute Syndrome Foundation and NORD Launch The Cute Syndrome Foundation Global SCN8A Survey Series

Posted at March 3, 2021 11:31 am by Lisa Sencen

The Cute Syndrome Foundation and the National Organization for Rare Disorders today launched a survey series to better understand patients with SCN8A mutations that cause epilepsy, movement disorders, behavioral challenges, and other medical complexities. SCN8A has been diagnosed in approximately 400 patients worldwide.

The new study, The Cute Syndrome Foundation Global… 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

February 2, 2021

TOPIC: Featured News, Research, #NORDintheNews

NORD Featured in February 2021 Issue of PharmaVOICE

Posted at February 2, 2021 10:25 am by Valaree DonFrancesco

In its February 2021 issue, PharmaVOICE features NORD in its Rare Disease Showcase column. In “Rare Disease: Tackling the Barriers to Rare Disease Treatment,” NORD’s Undiagnosed Patients Registry and the COVID-19 Community Survey Report are referenced. In “The State of Rare Disease, Diagnosis, and Treatment,” the Barriers to Rare Disease Diagnosis, Care and Treatment in the US: A 30-Year… Read More

January 26, 2021

TOPIC: Press Releases, Featured News, Patients & Members, Research, COVID-19

NORD and Partners Host FDA, CDC Leaders in Webinar Addressing Rare Disease Community on COVID-19 Vaccines

Posted at January 1, 2021 11:00 am by Valaree DonFrancesco

Washington, DC, January 262021In an effort to provide clear, usefulauthoritative information to rare disease patients and caregivers on the COVID-19 vaccineson January 15 the National Organization for Rare Disorders (NORD®), along with The ALS Association, Cystic Fibrosis Foundation and Muscular Dystrophy Association, hosted a special… Read More

December 16, 2020

TOPIC: Featured News, Research, Industry, Students for Rare

NORD Students for Rare Member Provides Her Perspective on “Unlocking the Power of Health Care Data” Summit Panel

Posted at December 12, 2020 08:38 am by Jennifer Ryan

As a 2020 scholarship recipient, I was able to attend the NORD Rare Diseases and Orphan Products and Breakthrough Summit in October and to hear from inspiring patients, hard-working medical professionals and research experts. Specifically, I was very drawn to the “Unlocking the Power of Health Care Data” panel, which discussed how data-driven health care is being used… Read More