A Phase 1b Trial to Assess the Pharmacokinetics of Ezutromid in Pediatric Duchenne Muscular Dystrophy Patients on a Balanced Diet
Francesco Muntoni(Great Ormond Street Hospital), Jonathon M. Tinsley(Mary Lyon Centre at MRC Harwell), Imelda Hughes(Manchester University NHS Foundation Trust), Helen Roper(Heart of England NHS Foundation Trust), Kay E. Davies(University of Oxford), Shawn Harriman(Amgen (United States)), Bina Tejura(Summit (United Kingdom)), Gary Layton(University of Geneva), Stefan Spinty(Alder Hey Children's NHS Foundation Trust)
Cited by 40
Related Papers
Function and Genetics of Dystrophin and Dystrophin-Related Proteins in Muscle
|Physiological Reviews|2002|1.2k
A gene (PEX) with homologies to endopeptidases is mutated in patients with X–linked hypophosphatemic rickets
|Nature Genetics|1995|1.1k
Improving genetic diagnosis in Mendelian disease with transcriptome sequencing
|Science Translational Medicine|2017|809