Therapies that are available and under development for Duchenne muscular dystrophy: What about lung function?
Gogou M, Pavlou E, Haidopoulou K. Therapies that are available and under development for Duchenne muscular dystrophy: What about lung…
The Dystrophinopathies
Thangarajh M. The Dystrophinopathies. Continuum (Minneapolis, Minn.) 2019;25:1619-1639 Abstract PURPOSE OF REVIEW: The dystrophinopathies are among the most common…
Muscular dystrophies
Mercuri E, Bonnemann CG, Muntoni F. Muscular dystrophies. Lancet (London, England) 2019;394:2025-2038 Abstract Muscular dystrophies are primary diseases of…
Caregiver preferences for emerging duchenne muscular dystrophy treatments: a comparison of best-worst scaling and conjoint analysis
Hollin IL, Peay HL, Bridges JF. Caregiver preferences for emerging duchenne muscular dystrophy treatments: a comparison of best-worst scaling and…
Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal muscular atrophy
Ke Q, Zhao ZY, Mendell JR, et al. Progress in treatment and newborn screening for Duchenne muscular dystrophy and spinal…
Duchenne muscular dystrophy: an historical treatment review
Werneck LC, Lorenzoni PJ, Ducci RD, Fustes OH, Kay CSK, Scola RH. Duchenne muscular dystrophy: an historical treatment review. Arq…
Muscle biopsies in clinical trials for Duchenne muscular dystrophy - Patients' and caregivers' perspective
Verhaart IEC, Johnson A, Thakrar S, et al. Muscle biopsies in clinical trials for Duchenne muscular dystrophy – Patients’ and…
Therapeutic developments for Duchenne muscular dystrophy
Verhaart IEC, Aartsma-Rus A. Therapeutic developments for Duchenne muscular dystrophy. Nature reviews. Neurology 2019;15:373-386 Abstract Duchenne muscular dystrophy (DMD)…
An overview of Compassionate Use Programs in the European Union member states
Balasubramanian G, Morampudi S, Chhabra P, Gowda A, Zomorodi B. An overview of Compassionate Use Programs in the European Union…
Precision Medicine and Exercise Therapy in Duchenne Muscular Dystrophy
Kostek M. Precision Medicine and Exercise Therapy in Duchenne Muscular Dystrophy. Sports (Basel, Switzerland) 2019;7 Abstract Precision medicine is…
