New treatments in spinal muscular atrophy: an overview of currently available data

@article{Ramdas2020NewTI,
  title={New treatments in spinal muscular atrophy: an overview of currently available data},
  author={Sithara Ramdas and Laurent Servais},
  journal={Expert Opinion on Pharmacotherapy},
  year={2020},
  volume={21},
  pages={307 - 315}
}
ABSTRACT Introduction: Spinal muscular atrophy (SMA) is one of the most common inherited neuromuscular disorders. It causes progressive muscle weakness and results in significant disability. Until recently, there were no drugs available for the treatment of SMA. Several phase 1–3 studies, including three double-blind randomized placebo-controlled studies have demonstrated the efficacy of disease-modifying approaches including gene replacement therapy, antisense oligonucleotides, and splicing… 
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Combination molecular therapies for spinal muscular atrophy: How much is enough?
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The neuromuscular field rejoices in the availability of now three treatments for this devastating disease, all of which clearly alter the natural history, and the question of how much fl SMN protein is “enough” is critical when one considers combining these medications.
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