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After previous reports that nearly all spinal muscular atrophy (SMA) patients treated with Zolgensma® achieved key motor milestones, Novartis announces publication of full SPR1NT results.
John Liddell, Chief Technologist at the Centre for Process Innovation (CPI), writes about the challenges associated with gene therapy products, which constitute a major portion of the overall cell and gene therapy market.
Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene therapy achieved motor milestones never observed in the natural history of spinal muscular atrophy.
Zolgensma, a one-off, potentially curative treatment for type 1 spinal muscular atrophy, is set to become the most expensive drug ever approved for use on the UK’s NHS.
In this article, we summarise the trial findings for the European Medicines Agency’s 13 most transformative therapies of 2020.
The EMA’s Pharmacovigilance Risk Assessment Committee (PRAC) began two new reviews and wrote guidance for healthcare professionals on the potential side effects of Strimvelis, Venclyxto and Zolgensma.
Treatment with Zolgensma® (onasemnogene abeparvovec) resulted in rapid and sustained improvement in motor function in spinal muscular atrophy (SMA) patients.
A summary of the CHMP meeting conclusions, including medicines recommended for approval and indication extension and several safety review findings.
