Roche's Muscular Atrophy Treatment Improves Motor Function In Pretreated Patients

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  • Roche Holdings AG RHHBY announced new two-year data from the JEWELFISH study evaluating Evrysdi (risdiplam) in people with Type 1, 2, or 3 spinal muscular atrophy (SMA) aged six months to 60 years.
  • Patients had been previously treated with other approved or investigational SMA-targeting therapies, including Biogen Inc's BIIB Spinraza (nusinersen) or Novartis AG's NVS Zolgensma (onasemnogene abeparvovec).
  • Data showed Evrysdi improved or maintained motor function and led to rapid increases in SMN protein levels sustained after two years of treatment.
  • Related: Roche Releases More Evrysdi Data For Very Young Infants With SMA
  • People with SMA cannot produce enough survival motor neuron (SMN) protein. 
  • The study showed Evrysdi led to a two-fold increase in median SMN protein levels versus baseline after four weeks of treatment in all patient groups, irrespective of previous treatment. 
  • The SMN protein levels achieved after four weeks of treatment were maintained for over two years. 
  • Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics Inc PTCT.
  • Price Action: RHHBY shares closed higher by 1.40% at $40.53 on Tuesday.
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