Methods
DMD
Data from placebo-treated ambulant males with DMD aged ≥ 5 years old with exon 51 skip amenable mutations recruited under NCT01254019 (provided by BioMarin Pharmaceutical Inc.) were included. These data were collected as part of a placebo-controlled double-blind randomized trial to assess the safety and efficacy of a novel therapy (drisapersen) for DMD; the clinical protocol inclusion criteria required patients be able to complete the 6MWT of > 75 m at each pre-drug visit. The follow-up period was 48 weeks from baseline (randomization) [The study protocol, informed consent, and other study documents were reviewed and approved by national, regional, or investigational center ethics committee or institutional review boards, as appropriate. The NCT01254019 study was performed in accordance with the International Conference on Harmonisation Good Clinical Practice guidelines, the Declaration of Helsinki (2008), and applicable country-specific requirements. Written informed consents from parents/caregivers and assent (from appropriately aged individuals) were obtained for all study participants prior to any study procedure.