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Safety and Efficacy of Givinostat for Patients with Muscular Dystrophy: A Systematic Review.
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Therapeutic approaches for Duchenne muscular dystrophy
Nature Reviews Drug Discovery, 2023Thomas C Roberts, Kay E Davies
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Golodirsen for Duchenne muscular dystrophy.
Drugs of Today, 2020Duchenne muscular dystrophy (DMD) is a life-shortening X-linked genetic disorder characterized by progressive wasting and weakening of muscles in boys. Loss-of-function mutations in the DMD gene, which codes for dystrophin, lead to this disease.
S. Anwar, T. Yokota
semanticscholar +4 more sources
Therapeutic developments for Duchenne muscular dystrophy
Nature Reviews Neurology, 2019Ingrid E C Verhaart +1 more
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Medical Journal of Australia, 1968
G. B. A. Duchenne's description of pseudohypertrophic muscular paralysis contains not only the first clear account of this disease, but also the first report of a practical instrument of muscle biopsy. Duchenne (1806-1875) was a French neurologist who was led to a careful study of neuromuscular diseases through his interest in the effects of ...
Irwin A. Brody, Robert H. Wilkins
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G. B. A. Duchenne's description of pseudohypertrophic muscular paralysis contains not only the first clear account of this disease, but also the first report of a practical instrument of muscle biopsy. Duchenne (1806-1875) was a French neurologist who was led to a careful study of neuromuscular diseases through his interest in the effects of ...
Irwin A. Brody, Robert H. Wilkins
openaire +6 more sources
BMJ, 2020
### What you need to know Duchenne muscular dystrophy (DMD) is a progressive and disabling neuromuscular condition that is often diagnosed late.1 In the UK the mean age of diagnosis has remained fairly static over the past 30 years, currently around 4.3 years of age.2 On average it takes 1.6 years from first parental concern to diagnosis of DMD,2 by ...
Luke Millington +3 more
openaire +2 more sources
### What you need to know Duchenne muscular dystrophy (DMD) is a progressive and disabling neuromuscular condition that is often diagnosed late.1 In the UK the mean age of diagnosis has remained fairly static over the past 30 years, currently around 4.3 years of age.2 On average it takes 1.6 years from first parental concern to diagnosis of DMD,2 by ...
Luke Millington +3 more
openaire +2 more sources
Current Opinion in Genetics & Development, 1991
Progress in understanding the role of dystrophin raises promising hopes for a treatment for Duchenne muscular dystrophy. In addition, great improvements have been made in the ability to diagnose this disease using simple molecular methods.
J. Wicki, J.T. Seto, J.S. Chamberlain
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Progress in understanding the role of dystrophin raises promising hopes for a treatment for Duchenne muscular dystrophy. In addition, great improvements have been made in the ability to diagnose this disease using simple molecular methods.
J. Wicki, J.T. Seto, J.S. Chamberlain
openaire +3 more sources