Children's Hospital Boston - Dr. Lou Kunkel Update

Posted by: Christine McSherry on January 30, 2013

My lab at Boston Children’s Hospital is working collaboratively with Pfizer to develop a small molecule treatment for Duchenne Muscular Dystrophy.  Our research involves testing FDA-approved drugs that target a pivotal pathway in the disease process for to find compounds that have the potential to prevent the disease onset and reverse the DMD phenotype.

A quote from Dr. Kunkel:

"Initial funding for this work was ending in the summer of 2012 and without support to bridge the gap until other funding sources became available, our progress would have halted.  Thankfully, through the Duchenne Dashboard, I was able to get funding within two months – an impossible time-frame if we had gone to a large foundation or government source.  Since the Duchenne Alliance-funded project began in August, we have screened more than 120 compounds and have already begun to test the best-acting compound from the zebrafish screens in a mouse model of DMD.  Since many of the Pfizer compounds being tested are in various stages of approval for human use, positive results could immediately be translated to the clinic.  This exciting and steady progress simply would not have been possible without funding from the Duchenne Alliance."

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