Nusinersen is currently commercialized under the brand name SPINRAZA in over 71 countries at the label-approved dose of 12 mg. “Continued progress to improve upon the remarkable initial ...
Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular atrophy (SMA) aged 2 to 17 years, who had previously been shut out of receiving ...
Novartis' OAV101 IT gene therapy showed positive results in Phase 3 trials for SMA, with data supporting its potential as a treatment option.
Cost-effectiveness body NICE has rejected Biogen’s Spinraza (nusinersen) therapy for the rare genetic muscle wasting disease spinal muscular atrophy (SMA) because of its high cost – but is in ...
Phase III Trial Shows Novartis’ Gene Therapy Significantly Improves Spinal Muscular Atrophy Outcomes
as well as motor function stabilization in patients who had discontinued Spinraza (nusinersen) or Evrysdi (risdiplam). 1 “In the STEER study evaluating treatment-naïve patients, OAV101 IT demonstrated ...
Treatment with Spinraza (nusinersen) can help stabilize or improve motor function in adults with SMA, according to a new ...
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Novartis Reports Updated Positive Data From Phase III SMA ProgramAdditionally, in the phase IIIb STRENGTH study, treatment with OAV101 IT demonstrated stabilization of motor function over 52 ...
treatment with OAV101 IT demonstrated stabilization of motor function over 52 weeks of follow-up in patients who had previously discontinued treatment with Spinraza (nusinersen) or Roche’s RHHBY ...
Evrysdi is the third treatment approved for SMA, an ultra-rare muscle wasting disease that can begin in early childhood, after Biogen’s Spinraza (nusinersen) and Novartis’ Zolgensma ...
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