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June 7, 2013

TOPIC: Patients & Members

What are Your Favorite Rare Disease Movies?

Posted by admin

Summer is right around the corner, which means hot days, grilling, more time outside, and of-course the inevitable rainy-day stuck inside. In preparation for those days, I am trying to compile a list of movies that could make up a “rare disease film festival.” (more…)

January 19, 2013

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

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

Posted by Steven A. Grossman

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