Ontology highlight
ABSTRACT:
SUBMITTER: Mercuri E
PROVIDER: S-EPMC7610147 | biostudies-literature | 2020 Apr
REPOSITORIES: biostudies-literature
Mercuri Eugenio E Muntoni Francesco F Osorio Andrés Nascimento AN Tulinius Már M Buccella Filippo F Morgenroth Lauren P LP Gordish-Dressman Heather H Jiang Joel J Trifillis Panayiota P Zhu Jin J Kristensen Allan A Santos Claudio L CL Henricson Erik K EK McDonald Craig M CM Desguerre Isabelle I
Journal of comparative effectiveness research 20200130 5
<b>Aim:</b> Strategic Targeting of Registries and International Database of Excellence (STRIDE) is an ongoing, multicenter registry providing real-world evidence regarding ataluren use in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). We examined the effectiveness of ataluren + standard of care (SoC) in the registry versus SoC alone in the Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS), DMD genotype-phenotype/-ataluren b ...[more]