Two years risdiplam: New data from SUNFISH, JEWELFISH in infants, children, adults with spinal muscular atrophy (SMA)

Roche announces new 2-year risdiplam data to

SUNFISH Part 1 showed risdiplam significantly improved motor function after 24 months of treatment in people aged 2-25 years with Types 2 or 3 SMA
JEWELFISH study preliminary 12 month data in previously treated patients showed rapid and sustained increases in SMN protein levels
Safety in SUNFISH and JEWELFISH was consistent with the safety profile observed to date and no new safety signals were identified

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Roche bringing patient support for spinal muscular atrophy (SMA) with Risdiplam

Spinraza: Biogen announced new results from the NURTURE study

  • After up to 4.8 years of continuous treatment with SPINRAZA, 100 percent of children treated pre-symptomatically were alive, and none require permanent ventilation
  • Patients continued to maintain and make progressive gains in motor function compared to the natural history of the disease, with 96 percent now able to walk with assistance
  • The NURTURE study was recently extended to evaluate the longer-term efficacy and safety of SPINRAZA up to 8 years of age
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