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Brogna C, Coratti G, Pane M, et al. Long-term natural history data in Duchenne muscular dystrophy ambulant patients with mutations…

Crudele JM, Chamberlain JS. AAV-based gene therapies for the muscular dystrophies. Human molecular genetics 2019;28:R102-R107   Abstract Muscular dystrophy is…

Atehortua SC, Lugo LH, Ceballos M, et al. Cost-Effectiveness Analysis of Diagnosis of Duchenne/Becker Muscular Dystrophy in Colombia. Value in…

de Moura MC, Wutzki HC, Voos MC, Resende MB, Reed UC, Hasue RH. Is functional dependence of Duchenne muscular dystrophy…

Larkindale J, Yang W, Hogan PF, et al. Cost of illness for neuromuscular diseases in the United States. Muscle &…

Pangalila RF, van den Bos GA, Bartels B, Bergen M, Stam HJ, Roebroeck ME. Prevalence of fatigue, pain, and affective…

Baiardini I, Minetti C, Bonifacino S, et al. Quality of life in Duchenne muscular dystrophy: the subjective impact on children…

Houwen-van Opstal SL, Jansen M, van Alfen N, de Groot IJ. Health-related quality of life and its relation to disease…

Teoh LJ, Geelhoed EA, Bayley K, Leonard H, Laing NG. Health care utilization and costs for children and adults with…

Thayer S, Bell C, McDonald CM. The Direct Cost of Managing a Rare Disease: Assessing Medical and Pharmacy Costs Associated…