Rare Disease News

Genzyme and NORD Establish Program to Help Undiagnosed Patients with Rare Diseases

Published December 16, 2013 by NORD

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CAMBRIDGE, MA and DANBURY, CT—-Genzyme, a Sanofi company, and the National Organization for Rare Disorders (NORD) today announced the creation of a fund to pay for standard diagnostic testing for …

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Orphan Drug Act at 30: Will Success Become Too Expensive?

Published January 19, 2013 by NORD

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Reprinted with permission from FDA Matters: By Steven Grossman The Orphan Drug Act (ODA) turned 30 this month, demonstrating that good laws really can have an enduring impact. Amidst the …

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NORD Issues Statement on the Death of Actor Jack Klugman

Published December 25, 2012 by NORD

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Jack Klugman touched the lives of millions of Americans through his support for what ultimately became the Orphan Drug Act. By portraying on his Quincy, M.E. television show the plight …

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Can IBM Watson Replace Dr. Dhaliwal? Probably Not!

Published December 6, 2012 by NORD

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Everyone in the rare disease community who watched IBM’s “Watson” computer perform on “Jeopardy” early this year probably started to wonder about the potential of “Watson-like” systems to assist doctors …

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Patients Will Benefit From New Public-Private Partnership

Published December 3, 2012 by NORD

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FDA Commissioner Margaret Hamburg, MD, today announced a new public-private partnership to develop regulatory science that will speed patient access to new medical device technologies. This is the first public-private …

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Glybera Becomes First-ever Gene Therapy Approved in Europe

Published November 2, 2012 by NORD

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According to a report on the BBC News web site today, the European Commission has — for the very first time anywhere in the Western world — given final approval …

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