New Developments with Prosensa and Sarepta for Exon Skipping in Duchenne

On Monday, the Today Show ran a segment about Jenn McNary and her two sons that have Duchenne, a deadly muscle disease that attacks primarily young boys.  Jenn had been passionate and effective in communicating the urgency of getting drugs for Duchenne approved by the FDA.   Jenn’s older son and thousands of boys like him need to  have the same hope that her younger son has who has been in a trial for the  exon skipping drug, etiplersen.  I applaud people and organizations that are willing to think outside the box in order to save their kids and the millions of other children that are suffering from fatal diseases.

On Friday, Prosensa filed for an IPO on NASDAQ.  Prosensa is an early pioneer in exon skipping for Duchenne and has partnered with Glaxo Smith Kline since 2009 to develop their lead compound, drisapersen.  We will watch closely to see the impact this has on additional drugs that Prosensa has in develoment.  We are very encouraged that the financial markets are taking notice of companies that develop drugs for Duchenne.  The expertise and resources that biotech and pharmaceutical companies bring to drug development will be crucial in order to commercialize Duchenne treatments.

This has been an exciting and hopeful time for families who are affected by Duchenne, and this weekend has shown how much promise exon skipping has for this devastating disease and how close we are to actually having  drugs to treat it.  CureDuchenne funded Prosensa in 2004 to develop their drugs for Duchenne.  And, in 2010 when AVI Biopharma, now Sarepta, was on clinical hold, CureDuchenne collaborated with Children’s National Medical Center and Foundation to Eradicate Duchenne to provide the funding neccessary for the company to do the studies which enabled them to progress to human clinical trials.

CureDuchenne will continue to think outside the box in order to get treatments to all the Duchenne patients that so desparately need them.  We are pround to be pioneers in exon skipping with both Sarepta and Prosensa and we expect that this disease will have treatments in the future.  The question is, how many of our sons must die before we get these drugs into our children?  A lot depends on aggressive thinking and funding…

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