Genetic and cell therapies - muscular dystrophy: the route to a cure
Koo T., Athanasopoulos T None., Dickson G None.
Duchenne muscular dystrophy (DMD) is one of the most common X‑linked and life‑threatening childhood diseases and affects about 1 in 3000 newborn boys. Lack of dystrophin protein causes severe progressive muscle wasting and death in the second/third decade of life, due to breathing and circulatory complications. Currently, there are no effective medications for DMD, but many different therapeutic approaches are under active development. In the case of genetic and cell therapies, pre‑clinical and clinical evaluations of safety and validity are paving the way towards effective new therapies which could be available routinely for DMD patients in the next 5 years.