Accelerating Access to Duchenne Drug Candidates

Yesterday Prosensa and CureDuchenne announced an extension of our collaboration and a path to provide access to four experimental drugs for Duchenne patients.

Ten years ago, CureDuchenne provided funding to Prosensa which allowed the company to move forward with their preclinical work and translate their exon skipping compounds into human clinical trials.  Since September 2013, our organization has worked extensively with Prosensa to design a plan so that the drisapersen trial participants as well as those boys that were in the PRO044/PRO45/PRO53 trials could have access to those drugs.

CureDuchenne, and its partners, will provide Prosensa with $7 million in funding, which will help the company to:

  • Commence dosing in a European Phase II clinical extension study of Prosensa’s Exon 44 skipping drug, PRO044;
  • Initiate a placebo-controlled clinical trial for PRO044 in the U.S. in the first half of 2015 (this drug could help up to six percent of those with Duchenne);
  • Re-initiate dosing for drisapersen clinical trial participants in North America and Europe and facilitate the drug’s NDA filing in the U.S. in 2014 (drisapersen could help up to 13 percent of the Duchenne population); and
  • Support the development of other exon skipping compounds, PRO045and/or PRO053(which could help up to eight percent of the Duchenne population respectively).

We have been pleased to see Prosensa progress with several compounds that will hopefully have a positive impact on Duchenne.  Drug development never goes quickly.  As a mother of a boy who could possibly benefit from PRO044, it has been painful and frustrating to see my son get weaker as we wait for a treatment.  It’s very likely that it will be too late for my son to benefit from this drug given his age, but we will hold out hope for all the boys that need to skip exons 44, 45, 51 and 53 that these drug will work and that they will be approved quickly.

These four drugs could potentially treat 35% of all Duchenne patients.  This is a perfect time for the Duchenne community to come together and help fund these programs as quickly as possible. We can hopefully, not only get these four drugs approved, but accelerate the development of therapies for the other 65% of Duchenne patients.  Go to to donate to this important project. Please contact our Cares team if you would like to help us raise the funds for these projects or learn more

In a perfect world, all boys with Duchenne would be cured now, all at the same time.  Unfortunately, different mutations will require different drugs.  CureDuchenne has funded all types of mutation research –  stopcodon mutations through PTC Therapeutic’s Ataluren (Translarna); duplication mutations through Dr. Kevin Flanigan at Nationwide Children’s Hospital; and exon 51 skipping with Prosensa’s drisapersen and Sarepta Therapeutics’ etiplersen.  We also are supporting research that is not mutation specific such as Akashi’s anti-fibrotic drug, ReveraGen’s non-hormonal steroid and Summit’s utrophin drug.  We will continue to work on one mutation, one exon…anything to save even one child at a time.  We believe that if we can get these first drugs approved, the other, rarer mutations will be approved much quicker.

I refuse to pop the champagne until we have approved drugs for Duchenne.  However, I am cautiously optimistic that we will have treatments for our children in the near future.  But, we can’t do it alone.  We need your help.  Everyone can do something, and all of us at CureDuchenne are here to help you personally impact this disease by working with you to create fundraising opportunities that are geared toward your area of interest.

All of us at CureDuchenne have been working on this project…working on getting access to drugs that can hopefully help our children, for a very long time.  I’d like to extend a big thank you to our small, nimble, but mighty CureDuchenne team:  Dr. Michael Kelly, Karen Harley, Drew Hoyer and Alicia Clough for juggling so many balls in the air as we created and implemented this program, and another, soon-to-be announced project.  A thanks also to our extended team, Sarah Burgess and Jennifer Wallace, as they have had such success while we have been knee-deep in finalizing this project.  Thanks also to the Duchenne families and donors who have supported us over the last 10 years.

We really need your help now.  Let’s be creative, let’s think outside the box…let’s fund this project together!

Our mission is our name…to cure Duchenne




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