A note from Debra

Pfizer Initiates Phase 2 Study of PF-06252616 in Duchenne Muscular Dystrophy

Posted on December 17, 2014 6:24 pm

Pfizer Inc. (NYSE:PFE) announced today enrollment of the first patient in a multicenter Phase II clinical trial of the investigational compound PF-06252616 in boys with Duchenne muscular dystrophy (DMD), a genetic disorder characterized by progressive muscle degeneration and weakness. PF-06252616 is an experimental, infused, anti-myostatin monoclonal antibody. Myostatin is a naturally occurring protein in muscles that helps control muscle growth; it is believed that blocking the activity of myostatin may have potential therapeutic application in treating muscle wasting diseases such as DMD.

“Our Duchenne community is proud to have the extraordinary passion and expertise of the Pfizer team fighting with us.”

“DMD is a devastating and debilitating disease impacting approximately 1 in 3,500 male births worldwide with no current treatment options,” said Kevin Lee, Ph.D., senior vice president and chief scientific officer of Pfizer’s Rare Disease Research Unit. “We are pleased to be taking this important next step in the development of PF-06252616 as an investigational therapy for DMD in the hopes of potentially bringing a much-needed therapy to individuals and families with this devastating disease.”

The phase 2 clinical trial will evaluate the safety, tolerability and efficacy of PF-06252616 in boys aged 6 to <10 years old diagnosed with DMD regardless of genotype. Based on the proposed mechanism of action of PF-06252616, Pfizer is exploring whether there is the potential to increase muscle mass and function in boys with DMD who are weak and have lost muscle.

Read more about this study in letter from Pfizer and frequently asked questions.

We are delighted to have Pfizer working on developing potential treatments to help find a cure for Duchenne.  We wish them much success in this trial.

Power Wheelchair Options for Duchenne Muscular Dystrophy Patients

Posted on November 20, 20145:03 pm

  Duchenne Therapy Network and CureDuchenne released their newest video blog that highlights power wheelchair options available for Duchenne muscular dystrophy patients.  Here we provide tips on what to look for when choosing a wheelchair. Front Wheel Drive Pros: Maneuverability Performance on terrain Space for foot movement Cons: Tracking issues at high speeds Mid-Wheel Drive Read more »


Posted on October 31, 201410:15 pm

  Last weekend, I had the honor, along with Dr. Mike Kelly, CureDuchenne’s Chief Scientific Officer, and other TACT members, to review Duchenne research projects. CureDuchenne was able to look out for the best interest of Duchenne patients by providing a scientific, patient organization and parent perspective.    The projects that we reviewed were: FibroGen Inc, Read more »