Biogen’s Salanersen Receives FDA Breakthrough Therapy Designation for Spinal Muscular Atrophy
Designation is supported by an exploratory analysis from the Phase 1b study showing that some children with SMA previously treated with gene therapy who had suboptimal clinical status experienced slowing of neurodegeneration and clinically meaningful improvements in motor function following initiation of salanersen Salanersen is an investigational antisense oligonucleotide dosed once-yearly with the potential to […]
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