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Archives for: October 5th, 2016

Rare Diseases & Orphan Products Breakthrough Summit Speakers

Written by Jennifer Huron on October 5, 2016
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RareSummit_Logo_RGB_smallWashington, D.C., October 5, 2016 – NORD, the leading independent nonprofit organization representing the 30 million Americans with rare diseases, today announced the three keynote speakers for its 2016 Rare Diseases & Orphan Products Breakthrough Summit to be held Oct. 17-18… Read More

Guest Blog: Tour For A Cure

Written by Jennifer Huron on August 3, 2016
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 By Alison Rockett Frase, Guest Blogger

In 1995 our son, Joshua, was born with a very rare muscle disorder called Myotubular Myopathy; an early death was imminent for our first-born. Through the despair and dismay, a vision of faith and hope was birthed through the Joshua Frase Foundation. Our vision seemed… Read More

A CRISPR Approach to Genome Editing

Written by Marsha Lanes on December 4, 2013
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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

Glybera Becomes First-ever Gene Therapy Approved in Europe

Written by E. Michael D. (Mike) Scott on November 2, 2012
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According to a report on the BBC News web site today, the European Commission has — for the very first time anywhere in the Western world — given final approval to market a gene therapy that corrects an inborn error of metabolism for the treatment of any type of… Read More