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December 6, 2012

TOPIC: Industry, Medical, Patients & Members, Research

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

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, Industry, Medical, Research

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

October 31, 2012

TOPIC: Medical, Patients & Members, Research

Creative Collaborations May Be Key to Reducing Chemotherapy Risks

Posted at October 10, 2012 01:33 pm by Marsha Lanes

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 to those in the rare disease community: (more…)