Rare Disease News

Informed Consent in Biobank Research

Published August 1, 2013 by NORD

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Genetic biobanks, storage facilities for DNA, genetic data and tissue samples, are becoming more prevalent in the rare disease community. The information and samples can be made available to approved …

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Supreme Court Decision Consistent With Longstanding NORD Position

Published June 14, 2013 by NORD

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Yesterday’s ruling by the U.S. Supreme Court that human genes are a product of nature and cannot be patented is being hailed by many as a victory for patients and …

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New Guidelines on Testing Kids’ DNA–the Cliff’s Notes Version

Published March 12, 2013 by NORD

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Exomes are big news. Sequencing of the protein-encoding part of the genome is increasingly solving medical mysteries in children.

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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Creative Collaborations May Be Key to Reducing Chemotherapy Risks

Published October 31, 2012 by NORD

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My friend’s 18-year-old son died recently following a 6-month battle with acute myeloid leukemia. There are many different stories I could tell about Tucker’s experience that are all too familiar …

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