Results 221 to 230 of about 39,769 (244)
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Current opinion in rheumatology, 1993
Advances in the understanding of the genetic basis for Duchenne muscular dystrophy over the past 4 years has led to the quick application of molecular diagnostics. More recently, attention has turned towards acquiring a better understanding of dystrophin biochemistry and the pathophysiologic consequences of dystrophin deficiency.
J R, Gorospe, E P, Hoffman
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Advances in the understanding of the genetic basis for Duchenne muscular dystrophy over the past 4 years has led to the quick application of molecular diagnostics. More recently, attention has turned towards acquiring a better understanding of dystrophin biochemistry and the pathophysiologic consequences of dystrophin deficiency.
J R, Gorospe, E P, Hoffman
openaire +1 more source
Therapeutic approaches for Duchenne muscular dystrophy
Nature Reviews Drug Discovery, 2023Thomas C Roberts, Kay E Davies
exaly
Duchenne Muscular Dystrophy (DMD) is a severe genetic disorder that affects the muscles, causing them to weaken over time. This condition primarily impacts boys and leads to progressive physical disability, requiring comprehensive care and management. This guide provides an accessible yet detailed explanation of DMD, addressing its causes, inheritance ...
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Induced pluripotent stem cells from a spinal muscular atrophy patient
Nature, 2008Allison D Ebert +2 more
exaly
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy
Nature Medicine, 2019Christopher E Nelson +2 more
exaly