Currently, there is one Duchenne-specific drug under investigation.
It works by allowing the protein machinery to read the instructions for making the protein past the stop-codon that occurs too early in the gene. This allows the gene to produce a functional version of the dystrophin protein.
Type of Drug
DMD Mutations Addressed
Current Development Phase
Oral medication taken three times daily
Phase 2 in <2 years of age
Phase 3 extension studies
Europe and Brazil for ambulatory boys 2yrs and older