Rare Disease News

Thoughtful Biosimilars Policy Is Best Way to Ensure Optimum Patient Outcomes

Published June 4, 2014 by NORD

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Biologic therapies have been lifesaving and life changing for the patients we represent – more than 30 million Americans with more than 7,000 rare diseases. Literally, hundreds of thousands of …

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VHL Alliance and NORD Launch New International Databank

Published May 14, 2014 by NORD

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To Support Research and Gain a Better Understanding of VHL and other Forms of Cancer, Patients and Medical Experts Around the World will Share Critical Information in Search of a …

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A CRISPR Approach to Genome Editing

Published December 4, 2013 by NORD

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Genome editing is a process that involves cutting out mutations in DNA and replacing them with new genetic material. This is an exciting idea because if it can be refined …

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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.

Privacy Protection in Whole Genome Sequencing

Published January 15, 2013 by NORD

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Whole genome sequencing (WGS) is the genetic test that determines the order of all 3 billion letters in a person’s DNA, and is a technology that has become well known …

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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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