Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients

Duchenne muscular dystrophy

(2021) Dongsheng Duan et al.   Nature Reviews Disease Primers

Life expectancy at birth in Duchenne muscular dystrophy: a systematic review and meta-analysis

(2020) Erik Landfeldt et al.   EUROPEAN JOURNAL OF EPIDEMIOLOGY

Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

(2020) Eugenio Mercuri et al.   Journal of Comparative Effectiveness Research

Cardiorespiratory progression over 5 years and role of corticosteroids in DMD: a single site retrospective longitudinal study

(2020) Federica Trucco et al.   CHEST

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

(2020) Craig Campbell et al.   Journal of Comparative Effectiveness Research

Ataluren use in patients with nonsense mutation Duchenne muscular dystrophy: patient demographics and characteristics from the STRIDE Registry

(2019) Francesco Muntoni et al.   Journal of Comparative Effectiveness Research

Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study

(2018) Craig M McDonald et al.   LANCET

Diagnosis and management of Duchenne muscular dystrophy, part 3: primary care, emergency management, psychosocial care, and transitions of care across the lifespan

(2018) David J Birnkrant et al.   LANCET NEUROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management

(2018) David J Birnkrant et al.   LANCET NEUROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management

(2018) David J Birnkrant et al.   LANCET NEUROLOGY

Longitudinal Pulmonary Function Testing Outcome Measures in Duchenne Muscular Dystrophy: Long-term Natural History with and without Glucocorticoids

(2018) Craig M. McDonald et al.   NEUROMUSCULAR DISORDERS

Ataluren in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial

(2017) Craig M McDonald et al.   LANCET

The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review

(2017) S. Ryder et al.   Orphanet Journal of Rare Diseases

Statistics in Brief: An Introduction to the Use of Propensity Scores

(2015) Maria C. S. Inacio et al.   CLINICAL ORTHOPAEDICS AND RELATED RESEARCH

The TREAT-NMD DMD Global Database: Analysis of More than 7,000 Duchenne Muscular Dystrophy Mutations

(2015) Catherine L. Bladen et al.   HUMAN MUTATION

A Community-Engaged Approach to Quantifying Caregiver Preferences for the Benefits and Risks of Emerging Therapies for Duchenne Muscular Dystrophy

(2014) Holly L. Peay et al.   CLINICAL THERAPEUTICS

Ataluren treatment of patients with nonsense mutation dystrophinopathy

(2014) Katharine Bushby et al.   MUSCLE & NERVE

The cooperative international neuromuscular research group duchenne natural history study-a longitudinal investigation in the era of glucocorticoid therapy: Design of protocol and the methods used

(2013) Craig M. McDonald et al.   MUSCLE & NERVE

The cooperative international neuromuscular research group Duchenne natural history study: Glucocorticoid treatment preserves clinically meaningful functional milestones and reduces rate of disease progression as measured by manual muscle testing and othe

(2013) Erik K. Henricson et al.   MUSCLE & NERVE

195th ENMC International Workshop: Newborn screening for Duchenne muscular dystrophy 14–16th December, 2012, Naarden, The Netherlands

(2013) Juliet A. Ellis et al.   NEUROMUSCULAR DISORDERS

Phase 2a Study of Ataluren-Mediated Dystrophin Production in Patients with Nonsense Mutation Duchenne Muscular Dystrophy

(2013) Richard S. Finkel et al.   PLoS One

Long-term benefits and adverse effects of intermittent versus daily glucocorticoids in boys with Duchenne muscular dystrophy

(2012) V. Ricotti et al.   JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY

Motor and respiratory heterogeneity in Duchenne patients: Implication for clinical trials

(2011) Véronique Humbertclaude et al.   EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management

(2009) Katharine Bushby et al.   LANCET NEUROLOGY

Diagnosis and management of Duchenne muscular dystrophy, part 2: implementation of multidisciplinary care

(2009) Katharine Bushby et al.   LANCET NEUROLOGY