CureDuchenne has worked closely with biotech and pharmaceutical companies for the past decade with a goal of accelerating treatments for Duchenne muscular dystrophy.  CureDuchenne was the first organization to partner with a biotech company to develop exon skipping to treat Duchenne almost ten years ago when we invested in Prosensa, a Dutch biotech company.  Since then, we have continued to work with GlaxoSmithKline, the large pharmaceutical company that licensed Prosensa’s drug, drisapersen.  Our support for exon skipping was extended when we funded the development of Sarepta Therapeutics’ drug, etiplersen.  We have continued to partner with multiple companies to develop a robust pipeline of potential drugs to treat DMD.

I believe that I have a unique perspective on our challenge. I have a son with Duchenne and know that we desperately need meaningful treatments as quickly as possible; but working with biotech and pharmaceutical companies over this past ten years has given me an insight into the challenges associated with the drug development and discovery process.

On September, 13 at 2:00pm ET, CureDuchenne is hosting a webinar for rare disease advocates that walk through the basic processes of drug development; from preclinical, to clinical and the approval process. (Click here to register:   Leading scientists from Pfizer, Shire and Genzyme will share their insight and experiences and hopefully offer clarity around what can seem to be a long and complex process to drug approval.

Parents and patients are able to submit questions in advance, and we will ask as many as we can.  Please note that the presenters will not be able to answer specific questions about a company, drug or disease.  You may submit your questions to

It is in everyone’s best interest for industry and the rare disease community to understand each other and have an open dialog.  As federal funding decreases, it is increasingly important to motivate and support our industry partners that will invest the hundreds of millions of dollars that it takes to bring a drug to patients.

To register for the webinar:

To submit questions for the webinar:



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