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November 4, 2021

TOPIC: Press Releases, Medical, Research

Committing to Care, Access, Equity and Research: NORD Announces 31 Rare Disease Centers of Excellence

Posted at November 11, 2021 08:30 am by Rohan Narayanan

National Organization for Rare Disorders® (NORD) provides new designation to medical centers to define standards for high-quality specialized care for rare disease patients

Washington, DCNovember 4, 2021— Today, the National Organization for Rare Disorders (NORD) announced 31 NORD Rare Disease Centers of Excellence across the United States, establishing a unique network of medical centers, clinics, and institutions to advance care and expand access for rare disease patients. The program is being led by NORD to promote outstanding treatment for rare disease patients regardless of disease or geography, elevate collaboration, improve standards of care, advance research, and… Read More

October 27, 2021

TOPIC: Press Releases, Research

NIH, FDA and 15 private organizations join forces to increase effective gene therapies for rare diseases

Posted at October 10, 2021 01:43 pm by Rohan Narayanan

October 27, 2021 – The National Institutes of Health, U.S. Food and Drug Administration, 10 pharmaceutical companies and five non-profit organizations have partnered to accelerate development of gene therapies for the 30 million Americans who suffer from a rare disease. While there are approximately 7,000 rare diseases, only two heritable diseases currently have FDA-approved gene therapies. The newly launched Read More

September 23, 2021

TOPIC: Press Releases, Research

NORD Welcomes Aliza Fink, D.Sc. as Director of Research Programs

Posted at September 9, 2021 10:24 am by Rohan Narayanan

Washington, DC, September 23, 2021—The National Organization for Rare Disorders (NORD®), the leading patient advocacy organization dedicated to the identification, treatment and cure of rare diseases, announced the appointment of Aliza Fink, D.Sc. as the new Director of Research Programs. Fink, an epidemiologist by trade, brings decades of expertise and proficiency to NORD’s ongoing efforts to identify real-world research,… Read More

September 22, 2021

TOPIC: Press Releases, Research, Registries

Tatton Brown Rahman Syndrome Community and NORD® Launch Natural History Study of Tatton Brown Rahman Syndrome (TBRS)

Posted at September 9, 2021 08:00 am by Rohan Narayanan

Research study is open to participants worldwide to advance understanding and treatments for TBRS, a mutation of DNMT3A, causing overgrowth, autism, intellectual disabilities, orthopedic concerns, cardiac issues, and a variety of other health and developmental concerns.

Stanfordville, New York, September 21 — Tatton Brown Rahman Syndrome Community and the National Organization for Rare Disorders, Inc. today launched the largest-ever study… Read More