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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