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Sarepta Therapeutics’ Orphan Drug Eteplirsen For Duchenne Muscular Dystrophy: The Human Side Of The Story

November 13, 2012

       The Orphan Druganaut Blog and other media are reporting on the business angle of bringing Sarepta Therapeutics’ orphan drug Eteplirsen for Duchenne Muscular Dystrophy to market. But there is the human side of the story behind these business headlines – the patient and their families, and the advocacy groups that work endlessly behind the scenes to bring drugs, treatments, and hope. 

        Jenn McNary is a mother of 6 from Vermont who has 2 boys with Duchenne Muscular Dystrophy (DMD). Only 1 son has access through a clinical trial to Eteplirsen. Jenn McNary has petitioned the FDA to give accelerated approval to Eteplirsen so that her second son and others will have access to this drug.

         ABC Good Morning America reports on this story.    


Orphan Druganaut November 6, 2012 Blog Post titled, “The Power Of Patient Advocacy Groups On Orphan Drug Development: Sarepta Therapeutics’ Orphan Drug Eteplirsen For Duchenne Muscular Dystrophy”   

YouTube October 25, 2012 posting of Video,”Q&A with Sarepta President & CEO, Chris Garabedian”  

Orphan Druganaut September 10, 2012 Blog Post titled, “The Human Side of an Orphan Drug”  

Cure Duchenne

Duchenne Muscular Dystrophy Research Fund

Foundation to Eradicate Duchenne (FED)

Muscular Dystrophy Association

Parent Project Muscular Dystrophy (PPMD)

The Duchenne Alliance

Copyright © 2012-2013, Orphan Druganaut Blog.  All rights reserved.

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