Key clinical point: Ataluren slowed functional decline in children with nonsense mutation Duchenne muscular dystrophy.
Major finding: Children with nonsense mutation DMD in the STRIDE Registry lost less distance (–35 meters) on the 6-minute walk test after 48 weeks taking ataluren than patients receiving ataluren in a previous randomized controlled trial (–42.2 meters) or those children receiving placebo (–57.6m) in the randomized controlled trial for ataluren.
Study details: The findings are based on outcomes from 184 real-world patients with nonsense mutation DMD who took ataluren, compared with 228 participants in a randomized controlled trial for ataluren.
Disclosures: The study and STRIDE Registry is funded by PTC Therapeutics with TREAT-NMD and the Cooperative International Neuromuscular Research Group. Mr. Delage and five other authors are employees of PTC Therapeutics, six authors had received speaker or consultancy fees or served on the advisory board of a variety of companies.
Delage A et al. AANEM 2019, Abstract 115.