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January 19, 2013

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

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

January 15, 2013

TOPIC: Patients & Members, Research

Privacy Protection in Whole Genome Sequencing

Posted at January 1, 2013 05:11 pm 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 14, 2012

TOPIC: Advocacy, Patients & Members

Campaign to Educate State Legislators for Rare Disease Day

Posted at December 12, 2012 08:34 pm 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!

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…)