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Published on: August 8, 2019
Triumphs, Trials, and Future Considerations in Genetic Therapies for Hereditary Neuromuscular Diseases
W David Arnold1, Kajri Majithia2
1Executive Director of NextGen Precision Health, and in the Department of Physical Medicine and Rehabilitation, University of Missouri-Columbia, Columbia, Missouri.
Abstract:
Neuromuscular diseases include conditions that affect the spinal motor neurons, peripheral nerves, neuromuscular junctions, and muscles, and they can result from acquired and inherited causes. The number of genetic therapies targeting the inherited causes of neuromuscular diseases has surged in the last decade. This review aims to highlight the current state of genetic therapies within the framework of precision medicine, focusing on the achievements and the gaps that remain. A major emphasis is on spinal muscular atrophy, Duchenne muscular dystrophy, and amyotrophic lateral sclerosis, as these neuromuscular diseases have seen tremendous recent advancements. We will also discuss the future considerations necessary to accelerate the development of next-generation genetic therapies and enhance therapeutic outcomes for patients with neuromuscular diseases.
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