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- W4313593882 abstract "Spinal muscular atrophy (SMA) is a rare neuromuscular condition, characterized by loss of motor neurons as a result of a mutation in the survival motor neuron gene. This results in muscle wasting and in the most common and severe type, death before 24 months. Over the recent years there has been a dynamic shift in the therapeutic options for these patients involving both approved therapies, including gene therapy, and access to clinical trials in genetic modifying therapies to indirectly improve the survival motor neuron protein level and hence strength, muscle promotor therapies, up/down regulation of modifier genes and SMN independent therapies. This review addresses the pathogenesis of SMA and the resultant therapies available as well as the current clinical trials and future development." @default.
- W4313593882 created "2023-01-06" @default.
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- W4313593882 date "2023-01-01" @default.
- W4313593882 modified "2023-09-27" @default.
- W4313593882 title "Therapeutic advances in spinal muscular atrophy" @default.
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- W4313593882 doi "https://doi.org/10.1016/j.paed.2022.12.010" @default.
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