Meta-analyses of ataluren randomized controlled trials in nonsense mutation Duchenne muscular dystrophy.

Conclusion: These meta-analyses support previous evidence for ataluren in slowing disease progression versus placebo in patients with nmDMD over 48 weeks. Treatment benefit was most evident in patients with a baseline 6MWD ≥300-<400 m (the ambulatory transition phase), thereby informing future trial design. PMID: 32851872 [PubMed - as supplied by publisher]
Source: Journal of Comparative Effectiveness Research - Category: General Medicine Tags: J Comp Eff Res Source Type: research