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Realising milestones with gene therapy for SMA
Novartis Gene Therapies' Vice President for Clinical Development & Analytics shares what shaped the success of one of the few commercially-available gene therapies.
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Spinal muscular atrophy (SMA)
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Novartis releases long-term data for SMA gene therapy
Long-term follow-up study data for Zolgensma®, Novartis’ one-time gene therapy for spinal muscular atrophy (SMA), has shown promising overall milestone achievements.
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Converging knowledge and technology to transform neuromuscular disease treatment
With the emergence of innovative therapeutic technologies and progress in the genetic understanding of diseases, Biogen’s Toby Ferguson explains why it is a promising time for the development of therapies for neuromuscular conditions such as amyotrophic lateral sclerosis (ALS).
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Final positive results of Zolgensma® trial for SMA published
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.
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Fund to fast-track innovative drugs to NHS
The UK Government has announced the launch of the Innovative Medicines Fund to bring cutting-edge treatment more quickly to NHS patients.
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Roche’s Evrysdi® shows long-term efficacy in spinal muscular atrophy
New data demonstrates the long-term safety and efficacy of Evrysdi® (risdiplam) in a broad population of people with spinal muscular atrophy (SMA).
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Roche awarded 2022 Drug Discovery of the Year for Evrysdi®
The development team behind Roche’s Evrysdi® (risdiplam), the first mRNA splicing modifier to be approved, have been honoured with the BPS’s Drug Discovery of the Year Award 2022.
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NICE recommends risdiplam for spinal muscular atrophy
The new draft guidance recommends Evrysdi (risdiplam) to treat spinal muscular atrophy as part of a managed access agreement (MAA).
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Babies with SMA able to swallow with Evrysdi® treatment
All five pre-symptomatic babies with spinal muscular atrophy (SMA) were able to swallow and feed orally after 12 months of treatment.
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Taking cell and gene therapies from idea to commercialisation
David Vincent, Process Development Lead at eXmoor Pharma, details some of the critical steps that form the manufacturing strategy for providers of cell and gene therapies.
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Collaboration to enable standardisation of AAV quality measurement
US Pharmacopeia to collaborate on assessment of analytical methods for adeno-associated viruses (AAVs) to facilitate the development of reference standards.
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Zolgensma® meaningfully improves spinal muscular atrophy in patients
Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene therapy achieved motor milestones never observed in the natural history of spinal muscular atrophy.
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Curative cell and gene therapies and healthcare system disruption
In this article, experts from Charles River Associates discuss how the introduction of cell and gene therapies will impact upon various aspects of the healthcare system, from provision of care to delivery and supply, and pricing and market access.
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Evrysdi™ approved for treatment of spinal muscular atrophy in Europe
The European Commission has granted marketing authorisation to Roche’s Evrysdi™ (risdiplam) as a treatment for spinal muscular atrophy in infants, children and adults.
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NICE approves gene therapy for 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.
