Duchenne muscular dystrophy (DMD) is a monogenic muscle-wasting disorder and a priority candidate for molecular and cellular therapeutics. Although rare, it is the most common inherited myopathy affecting children and so has been the focus of intense research activity. It is caused by mutations that disrupt production of the dystrophin protein, and a plethora of drug development approaches are under way that aim to restore dystrophin function. In this article the latest therapeutic strategies that are under development and being deployed to treat DMD are discusses. Lessons from drug development programmes are likely to have a major impact on the DMD field, but also on molecular and cellular medicine more generally. Read the full article here.
A rare presentation of Maffucci syndrome