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.
Drug Name | Type of Drug | DMD Mutations Addressed | Sponsor | Current Development Phase | Approval Status |
|---|---|---|---|---|---|
ataluren | Oral medication taken three times daily | Nonsense mutation | PTC Therapeutics | Phase 2 in <2 years of age Phase 3 extension studies | Europe and Brazil for ambulatory boys 2yrs and older |