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January 19, 2013

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

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

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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 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

January 15, 2013

TOPIC: Patients & Members, Research

Privacy Protection in Whole Genome Sequencing

Posted by Marsha Lanes

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 to the rare disease community.  It can reveal not only the genes responsible for production of an abnormal protein associated with a disease, but also the presence of other… Read More

December 25, 2012

TOPIC: Advocacy, Featured News, Industry

NORD Issues Statement on the Death of Actor Jack Klugman

Posted 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 14, 2012

TOPIC: Advocacy, Patients & Members

Campaign to Educate State Legislators for Rare Disease Day

Posted by admin

Each year NORD provides a list of suggested activities for Rare Disease Day. This year, one of those major projects is a nationwide campaign to help leaders of rare disease patient organizations — and those who support them — educate their state officials for Rare Disease Day 2013 (Feb. 28, 2013). Read More

December 6, 2012

TOPIC: Industry, Medical, Patients & Members, Research

Can IBM Watson Replace Dr. Dhaliwal? Probably Not!

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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 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