This information is brought to you by Annemieke Aartsma-Rus and adapted from Treat-NMD.
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.
Funding from Duchenne Parent Project (the Netherlands) and Duchenne Parent Project Onlus (Italy) for travel costs to allow Annemieke Aartsma-Rus to attend annual international meetings in the US and Italy is gratefully acknowledged.