Sharon Hesterlee's Blog – August 2012 Archive (3)

Interview with Chris Garabedian, President and CEO of Sarepta Therapeutics

We are all acutely aware of the need for an effective treatment for Duchenne. Finding a way for children and young men affected by Duchenne to lead longer, healthier lives is at the heart of all of our efforts as families’ fight this devastating disease.


This week PPMD received an…


Added by Sharon Hesterlee on August 31, 2012 at 10:30am — 2 Comments

Increasing stem cell survival in Duchenne therapy

One approach to improving muscle strength in Duchenne is to replace unhealthy muscle cells that lack dystrophin with donor stem cells that have normal levels of dystrophin. These cells might come from a relative unaffected by Duchenne or even a person with Duchenne’s own cell, after dystrophin is restored though a technique like gene therapy.


Although we’ve…


Added by Sharon Hesterlee on August 29, 2012 at 2:00pm — 6 Comments

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