The trial is aimed at assessing the safety, tolerability and efficacy of PF-06252616 in boys aged six to less than ten years old, diagnosed with Duchenne muscular dystrophy regardless of genotype.
Pfizer Rare Disease Research Unit senior vice-president and chief scientific officer, Kevin Lee, said, 'Duchenne muscular dystrophy is a devastating and debilitating disease impacting approximately 1 in 3,500 male births worldwide with no current treatment options. 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.'
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