Ataluren (Translarna) for Nonsense Mutation DMD
CompletedPhase 3Other Therapeutic Approaches
Study Overview
- Age
- 5–18 years
- Phase
- Phase 3
- Sponsor
- PTC Therapeutics
- Therapeutic Approach
- Other Therapeutic Approaches
- Variant Requirement
- Nonsense mutation in the DMD gene (premature stop codon)
- Eligible Sex
- Male
- Ambulation
- Ambulatory and Non-Ambulatory
- Countries
- United StatesCanada
Study Requirements and Criteria
Steroid Use
Background corticosteroid therapy allowed per standard of care
Inclusion Criteria
Male aged 5–18 years with nonsense mutation DMD. Ambulatory or non-ambulatory. Able to perform pulmonary function testing.
Exclusion Criteria
Amenable to exon skipping therapy. Severe cognitive impairment preventing cooperation. Uncontrolled seizure disorder.
Clinical Trial Registry
NCT ID
NCT03179631This information is provided for educational purposes only. Always consult the study investigators before making medical decisions.