Library Articles Archive • World Duchenne

Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-kappaB Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy

Finanger E, Vandenborne K, Finkel RS, et al. Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-kappaB Inhibitor, in Pediatric…

Analysis of motor and respiratory function in Duchenne muscular dystrophy patients

Luiz LC, Marson FAL, Bresciani Almeida CC, Toro A, Nucci A, Ribeiro JD. Analysis of motor and respiratory function in…

Echographic Assessment of Diaphragmatic Function in Duchenne Muscular Dystrophy from Childhood to Adulthood

Fayssoil A, Chaffaut C, Ogna A, et al. Echographic Assessment of Diaphragmatic Function in Duchenne Muscular Dystrophy from Childhood to…

Lower Extremity Functional Outcome Measures in Duchenne Muscular Dystrophy-A Delphi Survey

Senesac CR, Lott DJ, Willcocks RJ, Duong T, Smith BK. Lower Extremity Functional Outcome Measures in Duchenne Muscular Dystrophy-A Delphi…

Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification Methodology

Aartsma-Rus A, Morgan J, Lonkar P, et al. Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification Methodology. Journal…

Molecular genetic testing and diagnosis strategies for dystrophinopathies in the era of next generation sequencing

Zhang K, Yang X, Lin G, Han Y, Li J. Molecular genetic testing and diagnosis strategies for dystrophinopathies in the…

Management of Adrenal Insufficiency Risk After Long-term Systemic Glucocorticoid Therapy in Duchenne Muscular Dystrophy: Clinical Practice Recommendations

Bowden SA, Connolly AM, Kinnett K, Zeitler PS. Management of Adrenal Insufficiency Risk After Long-term Systemic Glucocorticoid Therapy in Duchenne…

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Phase 1 Study of Edasalonexent (CAT-1004), an Oral NF-kappaB Inhibitor, in Pediatric Patients with Duchenne Muscular Dystrophy

Analysis of motor and respiratory function in Duchenne muscular dystrophy patients

Echographic Assessment of Diaphragmatic Function in Duchenne Muscular Dystrophy from Childhood to Adulthood

Lower Extremity Functional Outcome Measures in Duchenne Muscular Dystrophy-A Delphi Survey

Report of a TREAT-NMD/World Duchenne Organisation Meeting on Dystrophin Quantification Methodology

Molecular genetic testing and diagnosis strategies for dystrophinopathies in the era of next generation sequencing

Management of Adrenal Insufficiency Risk After Long-term Systemic Glucocorticoid Therapy in Duchenne Muscular Dystrophy: Clinical Practice Recommendations

Muscle function and age are associated with loss of bone mineral density in Duchenne muscular dystrophy

Use of bone age for evaluating bone density in patients with Duchenne muscular dystrophy: a preliminary report

Overview of drisapersen (GSK2402968/PRO051) clinical trials

Road to independence: Documentary about young people with NMD

Webinar Recording: Understanding the role of dystrophin in the brain in DMD/BMD

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