Abstract
Childhood neuromuscular disorders are devastating conditions associated with major disability and shortened life span. They encompass common muscular dystrophies such as Duchenne muscular dystrophy (DMD); motorneuron diseases such as spinal muscular atrophy (SMA), and less common limb girdle muscular dystrophies and congenital myopathies. Each of these conditions presents its own therapeutic challenges. The major hurdle in muscular dystrophies is the abundance of the target organ (skeletal muscle is ~40% of the body weight), the need to target the heart and, in DMD, the large size of its cDNA that precludes its packaging in commonly used viral vectors.
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