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Five-year data from Genentech SMA study released
Spinal muscular atrophy (SMA) patients in the long-term extension Evrysdi study maintained or improved key developmental skills over the five-year period, data shows.
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Spinal muscular atrophy (SMA)
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New data revealed for Novartis SMA gene therapy
Novartis has revealed new data for its one-time gene therapy for spinal muscular atrophy (SMA) in older children.
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Year in review: EPR’s top stories of 2023
EPR wraps up the year with a selection of top stories from 2023, highlighting key trends and topics such as environmental monitoring (EM), quality (QA/QC), manufacturing, regulation and clinical development.
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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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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.
