Update in Duchenne and Becker muscular dystrophy
Waldrop MA, Flanigan KM. Update in Duchenne and Becker muscular dystrophy. Current opinion in neurology 2019;32:722-727 Abstract PURPOSE OF…
Share and protect our health data: an evidence based approach to rare disease patients' perspectives on data sharing and data protection - quantitative survey and recommendations
Courbier S, Dimond R, Bros-Facer V. Share and protect our health data: an evidence based approach to rare disease patients'…
Are there real benefits to implanting cardiac devices in patients with end-stage dilated dystrophinopathic cardiomyopathy? Review of literature and personal results
Palladino A, Papa AA, Morra S, et al. Are there real benefits to implanting cardiac devices in patients with end-stage…
Conservative gadolinium administration to patients with Duchenne muscular dystrophy: decreasing exposure, cost, and time, without change in medical management
Lang SM, Alsaied T, Moore RA, Rattan M, Ryan TD, Taylor MD. Conservative gadolinium administration to patients with Duchenne muscular…
Comprehensive Assessment of Cardiac Involvement in Muscular Dystrophies by Cardiac MR Imaging
Rochitte CE, Liberato G, Silva MC. Comprehensive Assessment of Cardiac Involvement in Muscular Dystrophies by Cardiac MR Imaging. Magnetic resonance…
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…
Making nutrition guidelines fit for purpose
Bero LA, Norris SL, Lawrence MA. Making nutrition guidelines fit for purpose. BMJ (Clinical research ed.) 2019;365:l1579 Introduction Dietary…
Duchenne muscular dystrophy: survival by cardio-respiratory interventions
Ishikawa Y, Miura T, Ishikawa Y, et al. Duchenne muscular dystrophy: survival by cardio-respiratory interventions. Neuromuscular disorders : NMD 2011;21:47-51…
Survival in Duchenne muscular dystrophy: improvements in life expectancy since 1967 and the impact of home nocturnal ventilation
Eagle M, Baudouin SV, Chandler C, Giddings DR, Bullock R, Bushby K. Survival in Duchenne muscular dystrophy: improvements in life…
The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence review
Ryder S, Leadley RM, Armstrong N, et al. The burden, epidemiology, costs and treatment for Duchenne muscular dystrophy: an evidence…
