Duchenne muscular dystrophy is the most common muscular dystrophy affecting children, due to loss of function mutations in the DMD gene. In addition to causing progressive muscle weakness, DMD is also associated with brain comorbidities, which affect nearly 50% of the affected boys.
A number of different therapeutic strategies are under development to improve outcome in DMD, some having received conditional approval in some countries, others at different stages of development.
In this lecture I will provide an overview of the status of some of the most promising therapies under development to improve muscle function in DMD. I will also present the work that our consortium (BIND Consortium, www.bindproject.eu) is doing to consider the implication of genetic therapies to also address the deficiency of the defective gene in brain.