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June 4, 2014

TOPIC: Medical, Patients & Members, Research

Thoughtful Biosimilars Policy Is Best Way to Ensure Optimum Patient Outcomes

Posted at June 6, 2014 01:47 pm by

Biologic therapies have been lifesaving and life changing for the patients we represent – more than 30 million Americans with more than 7,000 rare diseases. Literally, hundreds of thousands of rare disease patients have found their lives bettered by biologics and we are collectively grateful to the innovators and companies that have made this possible. Read More

December 4, 2013

TOPIC: Medical, Research

A CRISPR Approach to Genome Editing

Posted at December 12, 2013 04:30 pm by Marsha Lanes

Genome editing is a process that involves cutting out mutations in DNA and replacing them with new genetic material.  This is an exciting idea because if it can be refined to a level that is safe and effective, the prospect of eradicating genetic diseases is no longer science fiction.  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