Showing 473 results
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Ad hoc release /Zolgensma® (onasemnogene abeparvovec), a one-time administered gene therapy, has been recommended for European Commission (EC) conditional approval for patients with spinal muscular atrophy (SMA) and…
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Press release /Children with three copies of SMN2 treated presymptomatically achieved age-appropriate milestones, including standing and walking; required no ventilatory or feeding tube support; and had no serious…
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Press release /Kisqali plus an oral endocrine partner demonstrated significant efficacy with sustained benefit of nearly two years (median PFS 23.8 vs 13.0 months for endocrine therapy alone) and…
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Press release /Nearly two-thirds of patients (65.6%) in STR1VE-EU have already achieved developmental motor milestones not observed in the natural history of SMA Type 1 at a mean duration of follow-up of 10.6…
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Press release /Continuous Kesimpta® treatment for up to six years showed sustained efficacy in recently diagnosed (≤3 years) treatment-naïve people living with relapsing multiple sclerosis (RMS) in an analysis…
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Press release /Breakthrough Therapy Designation based on Phase III study showing the three-year relapse-free survival (RFS) rate for patients treated with the combination was 58%, compared to 39% with placebo1…
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Press release /CMLPath to Care(TM) collaboration replaces Glivec International Patient Access Program (GIPAP) with a new, independent, patient-centered access model GIPAP was introduced 15 years ago and has…
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Press release /Priority Review designation based on Phase III study showing 53% reduction in risk of recurrence or death with Tafinlar + Mekinist, as compared to placebo[1] Improvements observed in key…
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Press release /Priority Review based on Phase III study results showing 16.6 month median progression-free survival in previously untreated ALK+ metastatic NSCLC patients on Zykadia vs. 8.1 months treated with…
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