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The Promise and Pitfalls of AAV-Mediated Gene Therapy for Duchenne Muscular Dystrophy. [PDF]
Curr Issues Mol BiolKurshakova EV +3 more
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Exploring the Role of Telemedicine in Duchenne Muscular Dystrophy: Benefits and Challenges. [PDF]
JMIR Form ResWasilewska E +10 more
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Optical genome mapping identifies a balanced inversion disrupting DMD in a patient with Duchenne muscular dystrophy. [PDF]
Mol CytogenetTurtinen T +8 more
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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 ...
Hannah, Fox +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 ...
Hannah, Fox +3 more
openaire +2 more sources
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 ...
I A, Brody, R H, Wikins
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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 ...
I A, Brody, R H, Wikins
openaire +4 more sources
Current Treatment Options in Neurology, 2001
Duchenne muscular dystrophy (DMD) is not treatable; there is no cure. More than a decade ago, randomized trials demonstrated that oral steroid therapy was of benefit to DMD patients by prolonging ambulation. Although few significant side effects were reported, study patients were followed for 18 months or less. However, when treating DMD with steroids,
Susan T., Iannaccone, Zohair, Nanjiani
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Duchenne muscular dystrophy (DMD) is not treatable; there is no cure. More than a decade ago, randomized trials demonstrated that oral steroid therapy was of benefit to DMD patients by prolonging ambulation. Although few significant side effects were reported, study patients were followed for 18 months or less. However, when treating DMD with steroids,
Susan T., Iannaccone, Zohair, Nanjiani
openaire +2 more sources
BMJ, 2010
I was diagnosed with Duchenne muscular dystrophy at the age of 3. My parents were always honest about my disease, but I didn’t really care much about it. As the progression was slow, I gradually began to understand its impact, including the physical restrictions. I’m now severely disabled and have lived much longer than the doctors expected.
Spies, Stefan +3 more
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I was diagnosed with Duchenne muscular dystrophy at the age of 3. My parents were always honest about my disease, but I didn’t really care much about it. As the progression was slow, I gradually began to understand its impact, including the physical restrictions. I’m now severely disabled and have lived much longer than the doctors expected.
Spies, Stefan +3 more
openaire +3 more sources
Neurology India, 2008
AbstractDuchenne muscular dystrophy, an X‐linked disorder, has an incidence of one in 5000 boys and presents in early childhood with proximal muscle weakness. Untreated boys become wheelchair bound by the age of 12 years and die of cardiorespiratory complications in their late teens to early 20s.
Yiu, Eppie M., Kornberg, Andrew J.
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AbstractDuchenne muscular dystrophy, an X‐linked disorder, has an incidence of one in 5000 boys and presents in early childhood with proximal muscle weakness. Untreated boys become wheelchair bound by the age of 12 years and die of cardiorespiratory complications in their late teens to early 20s.
Yiu, Eppie M., Kornberg, Andrew J.
openaire +3 more sources