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August 19, 2013

TOPIC: Patients & Members

The Power of Words

Posted at August 8, 2013 04:17 pm by

Quotes, mottos, and poems can be a source of motivation for many people.  Speeches are often capped off with a quote from a retired world leader, actress, writer, or extraordinary mind. Usually we don’t know the context in which these words were spoken, but being removed from the quotes gives us some flexibility in our personal interpretation. Read More

August 1, 2013

TOPIC: Medical, Patients & Members, Research

Informed Consent in Biobank Research

Posted at August 8, 2013 06:33 pm by Marsha Lanes

Genetic biobanks, storage facilities for DNA, genetic data and tissue samples, are becoming more prevalent in the rare disease community.   The information and samples can be made available to approved researchers for clinical studies, but unlike clinical trials, there is usually no personal benefit or follow-up provided to participants.  (more…)Read More

June 7, 2013

TOPIC: Patients & Members

What are Your Favorite Rare Disease Movies?

Posted at June 6, 2013 04:53 pm 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 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