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December 25, 2012

TOPIC: Featured News, Advocacy, Industry

NORD Issues Statement on the Death of Actor Jack Klugman

Posted at December 12, 2012 06:41 pm by Mary Dunkle

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 of people with rare diseases with no treatment, Mr. Klugman focused a spotlight on the need for incentives to encourage the development of therapies for small patient populations.  He… Read More

December 6, 2012

TOPIC: Medical, Patients & Members, Research, Industry

Can IBM Watson Replace Dr. Dhaliwal? Probably Not!

Posted at December 12, 2012 03:28 am by

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 in accelerating the accurate diagnosis of rare and unusual medical disorders — one of the first and most critical steps in assuring quality of care. … READ… Read More

December 3, 2012

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

Patients Will Benefit From New Public-Private Partnership

Posted at December 12, 2012 09:53 pm by

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 partnership specifically focusing on this need. (more…)

November 2, 2012

TOPIC: Featured News, Medical, Research, Industry

Glybera Becomes First-ever Gene Therapy Approved in Europe

Posted at November 11, 2012 07:37 pm by

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 to market a gene therapy that corrects an inborn error of metabolism for the treatment of any type of human genetic disorder. Read More