Nonsense Mutations Therapies
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Nonsense Mutations Therapies
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 |
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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 |