Risdiplam: First Approval

@article{Dhillon2020RisdiplamFA,
  title={Risdiplam: First Approval},
  author={Sohita Dhillon},
  journal={Drugs},
  year={2020},
  volume={80},
  pages={1853 - 1858}
}
Risdiplam (Evrysdi™) is an orally administered, survival motor neuron 2 (SMN2)-directed RNA splicing modifier being developed by Roche, PTC Therapeutics Inc and the SMA Foundation for the treatment of the spinal muscular atrophy. The small molecule is designed to treat spinal muscular atrophy caused by mutations in chromosome 5q leading to SMN protein deficiency. The drug boosts the ability of an alternative gene SMN2 to produce full-length and functional SMN protein. In August 2020, Evrysdi… 
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PTC announces the acceptance of the European Marketing Authorization application for EvrysdiTM (risdiplam) for the treatment of spinal muscular atrophy [media release
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