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Spinal muscular atrophy (SMA)
Long-term follow-up study data for Zolgensma®, Novartis’ one-time gene therapy for spinal muscular atrophy (SMA), has shown promising overall milestone achievements.
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).
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.
New data demonstrates the long-term safety and efficacy of Evrysdi® (risdiplam) in a broad population of people with spinal muscular atrophy (SMA).
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.
The new draft guidance recommends Evrysdi (risdiplam) to treat spinal muscular atrophy as part of a managed access agreement (MAA).
All five pre-symptomatic babies with spinal muscular atrophy (SMA) were able to swallow and feed orally after 12 months of treatment.
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.
US Pharmacopeia to collaborate on assessment of analytical methods for adeno-associated viruses (AAVs) to facilitate the development of reference standards.
Asymptomatic and symptomatic children treated with Zolgensma® (onasemnogene abeparvovec) gene therapy achieved motor milestones never observed in the natural history of spinal muscular atrophy.
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.
The European Commission has granted marketing authorisation to Roche’s Evrysdi™ (risdiplam) as a treatment for spinal muscular atrophy in infants, children and adults.
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.