We are thrilled to be working with amazing collaborators to help understand the burden of Duchenne muscular dystrophy (DMD). Here is some of our recent research published in the Orphanet Journal of Rare Diseases: The clinical course of Duchenne muscular dystrophy in the corticosteroid treatment era: a systematic literature review.
This work is the first comprehensive synthesis of literature describing the natural history of Duchenne muscular dystrophy (DMD) in the corticosteroid era in North America.
We were interested in understanding the age at the occurrence of the key clinical milestones that occur among those with DMD including loss of ambulation, the need for ventilation to support breathing and the development of cardiomyopathy. This work has also leant insight into a number of challenges in the interpretation and comparison of estimates of outcomes across studies; in particular, because of the highly heterogeneous clinical courses experienced by boys with DMD.