This research article, published in the Journal of Neuromuscular Diseases, provides a review of the impact of timing of clinical interventions in patients with Duchenne muscular dystrophy (DMD)
This research article, published in PLoS ONE, provides a summary of the prognostic indicators of disease progression in Duchenne muscular dystrophy (DMD)
This research article, published in the Journal of Comparative Effectiveness Research, provides results from a retrospective real-world analysis of corticosteroid use in patients with Duchenne muscular dystrophy (DMD)
In this video, recorded in 2022 for the Video Journal of Biomedicine, 2 authors of a research article present the background and findings from their study of a mutation-specific treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD)
This research article, published in the Journal of Comparative Effectiveness Research, provides results of meta-analyses comparing the efficacy of different corticosteroids in the management of nonsense mutation Duchenne muscular dystrophy (nmDMD)
This research article, published in Future Neurology, describes the symptoms of nonsense mutation Duchenne muscular dystrophy (nmDMD), and the impact of the condition on caregivers, at different stages of the disease
This case study, published in Acta Myologica, details an ambulant 2-year-old patient with nonsense mutation Duchenne muscular dystrophy (nmDMD), who received early treatment with a mutation-specific therapy in an expanded-access program
This research article, published in the Journal of Patient Reported Outcomes, describes the development of a conceptual model of the symptoms and impacts of nonsense mutation Duchenne muscular dystrophy (nmDMD), based on qualitative interviews with caregivers
This article, published in PharmacoEconomics Open, provides results from an investigation to determine the cost of illness of Duchenne muscular dystrophy (DMD)
This research article, published in the Journal of Patient Reported Outcomes, describes the development of a conceptual model of the impact of nonsense mutation Duchenne muscular dystrophy (nmDMD) on caregivers, based on interviews with parents of patients with the condition
This research article, published in the Journal of Comparative Effectiveness Research, provides results from a study investigating the efficacy of a mutation-specific therapy in delaying loss of ambulation (LoA) and respiratory function in patients with nonsense mutation DMD (nmDMD)
This research article, published in Muscle and Nerve, describes investigations into a well-developed analytical procedure for evaluating longitudinal treatment effects using NSAA data for illustration
This research article, published in Drug Metabolism and Pharmacokinetics, evaluates the in vitro and in vivo stability of ataluren-O-1β-acyl glucuronide metabolite
This research article, published in Pharmacology Research & Perspectives, investigates the plasma pharmacokinetics, metabolite profiles and their quantitative exposures in humans following a single oral dose of deflazacort
This research article, published in Pharmacology Research & Perspectives, evaluates CYP- and transporter-mediated drug interaction potentials of 6β-OH-21-desDFZ
This research article, published in Drug Metabolism and Disposition, looks at the in vivo metabolism of ataluren in selected species
This research article, published in Pharmacology Research and Perspectives, describes the in vitro metabolism and enzyme kinetics of ataluren and its interaction with CYP enzymes
This research letter, published in the JAMA Network Open, presents an analysis of two randomized, double-blind, placebo-controlled trials of a mutation-specific DMD gene therapy
This article, published in the Journal of Clinical Medicine, evaluates the developmental trajectory of working memory in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD)
This research article, published in the Journal of Comparative Effectiveness Research, examines the totality of efficacy evidence for a mutation-specific therapy in patients with nmDMD by meta-analysis
This research article, published in the Journal of Comparative Effectiveness Research, outlines the safety and effectiveness of a mutation-specific Duchenne muscular dystrophy (DMD) therapy in two propensity score-matched DMD patient populations
This clinical research article, published in the Muscle & Nerve Journal, compares disease progression among boys enrolled in the placebo arms of two DMD clinical trials receiving deflazacort or prednisone/prednisolone
This research article, published in the Journal of Comparative Effectiveness Research, outlines the initial demographics and baseline characteristics of patients enrolled in STRIDE, an observational registry of nmDMD patients receiving a mutation-specific DMD therapy
This article, published in the Neurology Journal, outlines the relationship between deficit in digit span and genotype in nmDMD.
This research article, published in the Muscle & Nerve Journal, details a post hoc analysis of the placebo arm of the Ataluren Confirmatory Trial (ACT) Duchenne muscular dystrophy (DMD) study
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Learn more about our phase I–III clinical trials, patient registries and natural history studies in DMD.