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December 16, 2013

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

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

Posted at December 12, 2013 08:39 pm by Mary Dunkle

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 people with mysterious, undiagnosed medical conditions.  The fund will help those who have applied to the National Institutes of Health (NIH) Undiagnosed Diseases Program but who cannot afford the… Read More

January 19, 2013

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

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

Posted at January 1, 2013 10:58 pm by

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 celebrations, a reporter asked me a provocative question:  Can we afford more orphan drugs costing hundreds of thousands of dollars per year?  FDA Matters answered “yes”…. Read More

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…)