Understanding Myostatin Inhibition (November 2015)

On November 11, 2015 PPMD hosted a webinar about the importance of myostatin inhibition research. Sponsored by educational partners Bristol-Myers Squibb and Pfizer, this webinar helped to put context and clarity around this treatment pathway so families have a better understanding as they see clinical trials in this area. Some years ago, researchers identified the myostatin pathway as a negative regulator of muscle growth. They isolated the cause of this disparity to a mutation in the gene that codes for the production of a hormone called myostatin, which tends to limit muscle growth. Scientists searching for a treatment theorize that inhibiting myostatin in boys with Duchenne will cause them to develop more muscle mass initially. Ideally, this surplus will offset the muscle loss associated with Duchenne, allowing boys to retain their ability to function for a longer period of time.