Stem cell-based therapies for Duchenne muscular dystrophy
Sun C, Serra C, Lee G, Wagner KR. Stem cell-based therapies for Duchenne muscular dystrophy. Experimental neurology 2020;323:113086 Abstract…
Evaluating the "return on patient engagement initiatives" in medicines research and development: A literature review
Vat LE, Finlay T, Jan Schuitmaker-Warnaar T, et al. Evaluating the "return on patient engagement initiatives" in medicines research and…
Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet database
Nguengang Wakap S, Lambert DM, Olry A, et al. Estimating cumulative point prevalence of rare diseases: analysis of the Orphanet…
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…
Ankle bracing practices in ambulatory, corticosteroid-naive boys with Duchenne muscular dystrophy
Kern V, Wicklund M, Haulman A, et al. Ankle bracing practices in ambulatory, corticosteroid-naive boys with Duchenne muscular dystrophy. Muscle…
Longitudinal natural history in young boys with Duchenne muscular dystrophy
Coratti G, Brogna C, Norcia G, et al. Longitudinal natural history in young boys with Duchenne muscular dystrophy. Neuromuscular Disord…
Influence of beta2 adrenergic receptor genotype on risk of nocturnal ventilation in patients with Duchenne muscular dystrophy
Kelley EF, Cross TJ, Snyder EM, McDonald CM, Hoffman EP, Bello L. Influence of beta2 adrenergic receptor genotype on risk…
Proceedings of a Parent Project Muscular Dystrophy Bone Health Workshop: Morbidity due to osteoporosis in DMD: The Path Forward May 12-13, 2016, Bethesda, Maryland, USA
Ward LM, Kinnett K, Bonewald L. Proceedings of a Parent Project Muscular Dystrophy Bone Health Workshop: Morbidity due to osteoporosis…
Relationships between DMD mutations and neurodevelopment in dystrophinopathy
Thangarajh M, Hendriksen J, McDermott MP, Martens W, Hart KA, Griggs RC. Relationships between DMD mutations and neurodevelopment in dystrophinopathy.…
Assisted standing for Duchenne muscular dystrophy
Pedlow K, McDonough S, Lennon S, Kerr C, Bradbury I. Assisted standing for Duchenne muscular dystrophy. Cochrane Db Syst Rev…
