- Translation update!
The aim of this overview is to inform patients and parents about the different therapeutic approaches for Duchenne muscular dystrophy currently under investigation, to describe the advantages and disadvantages of each approach and to list the hurdles that have to be overcome before these approaches can be applied to patients.
Note: The scientific field of Duchenne therapies is huge and rapidly evolving, making it impossible to include each and every approach in a brief overview. Thus, a general overview of the main approaches is provided.
This overview has last been updated in July 2016.
There are numerous therapeutic approaches in development and the majority follow the same road of preclinical to clinical studies. First the approach is tested in cultured patient cells, then in animal models of the disease (generally the mdx mouse) and then – when the results in cells and animals are sufficiently convincing, in patients (clinical trials). This part of therapeutic development can take a very long time as often unforeseen hurdles arise, which have to be overcome. Clinical trials are divided in two phases: the early phase trials are primarily done to show that the approach is safe, while in later stage trials the goal is to show that the treatment is efficient as well as safe. It is good to bear in mind that while each step is critical, the next step is always more challenging. So if a compound works in an initial step (e.g. cell studies) this is no guarantee for further successes in studies in animals and humans.