Research Archives - NORD (National Organization for Rare Disorders)

August 31, 2017

TOPIC: Events, Featured News, Industry, Medical, Patients & Members, Research

FDA Announces First Gene Therapy in U.S.

Posted at August 8, 2017 12:39 pm by Mary Dunkle

In an action the rare disease community has been eagerly awaiting, the Food and Drug Administration (FDA) has announced the first-ever approval of a gene therapy in the U.S. The product is Kymriah, and it is approved to treat certain children and young adults who have a cancer of blood and bone marrow known as acute lymphoblastic leukemia (ALL).

Approximately… Read More

Tags: David Lebwohl MD, Dr. Scott Gottlieb, FDA, Food and Drug Administration, Gene therapy, Next-Generation Treatments & Advancing Clinical Trials, NORD Rare Summit, Rare Diseases and Orphan Products Breakthrough Summit

July 25, 2017

TOPIC: Featured News, Medical, Patients & Members, Research

NORD Publishes New Rare Disease Report on Retinal Vasculopathy with Cerebral Leukodystrophy (RVCL)

Posted at July 7, 2017 03:41 pm by Christina Jensen

Washington, D.C., July 25, 2017—As part of its ongoing series to promote awareness and education regarding rare diseases, the National Organization for Rare Disorders (NORD) has published a report on Retinal Vasculopathy with Cerebral Leukodystrophy (RVCL). This new resource is available free online to individuals around the world.

As the primary advocacy organization in the U.S. for people who have… Read More

Tags: Dr. John Atkinson, Dr. M Kathryn Liszewski, Genetic Counselor, Marsha Lanes, Rare Disease Database, Rare Disease Report, rare genetic disease, research, Retinal Vasculopathy with Cerebral Leukodystrophy, RVCL, Washington University School of Medicine

July 20, 2017

TOPIC: Events, Featured News, Get Involved, Industry, Medical, Research

NORD Announces Keynote Speakers for 2017 Rare Diseases and Orphan Products Breakthrough Summit

Posted at July 7, 2017 09:54 am by Christina Jensen

Washington, D.C. July 19, 2017 – The National Organization for Rare Disorders (NORD), the leading independent nonprofit organization representing the 30 million Americans with rare diseases, has announced two keynote speakers for its 2017 Rare Diseases and Orphan Products Breakthrough Summit, to be held October 16-17 in Washington, D.C.

The meeting, which takes place on an annual basis, brings… Read More

Tags: Data-Sharing, Dr. Scott Gottlieb, FDA, FDA Commissioner, Keynote Address, Mike Porath, NIH, NORD Summit, Patient Assistance Programs, Rare Disease Summit, Rare Diseases and Orphan Products, Rare Diseases and Orphan Products Breakthrough Summit, Right to Try, The Mighty, TheMighty.com

July 7, 2017

TOPIC: Featured News, Medical, Research

NORD Publishes New Rare Disease Report on Bosma arhinia microphthalmia (BAM) syndrome

Posted at July 7, 2017 05:10 pm by Christina Jensen

Washington, D.C., July 7, 2017—As part of its ongoing series to promote awareness and education regarding rare diseases, the National Organization for Rare Disorders (NORD) has published a report on Bosma arhinia microphthalmia (BAM) syndrome. This new resource is available free online to individuals around the world.

As the primary advocacy organization in the U.S. for people who have rare… Read More

Tags: BAM Syndrome, Bosma arhinia microphthalmia (BAM) syndrome, Dr. John Graham, Dr. Natalie Shaw, National Institute of Environmental Health Sciences, National Institutes of Health, NIH, Rare Disease Database, Rare Disease Report, UCLA

June 13, 2017

TOPIC: Medical, Press Releases, Research, Sticky Posts for Clinicians and Researchers

NORD Publishes New Rare Disease Report on CARD9 Deficiency

Posted at June 6, 2017 09:28 am by Christina Jensen

Washington, D.C., June 13, 2017—As part of its ongoing series to promote awareness and education regarding rare diseases, the National Organization for Rare Disorders (NORD) has published a report on CARD9 Deficiency. This new resource is available free online to individuals around the world.

As the primary advocacy organization in the U.S. for people who have rare diseases, NORD provides… Read More

Tags: CARD9 Deficiency, Dr. Donald C. Vinh, Marsha Lanes, McGill University Health Centre Research Institute, Melanie Langelier RN, Rare Disease Reports

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