News

The purpose of SMA Europe is to provide a framework to stimulate collaboration and accelerate translational research pathways in SMA and promote patient care.

  1. AveXis’ name is changed to Novartis Gene Therapies EU Ltd

    AveXis, which has been part of Novartis since 2018, is becoming Novartis Gene Therapies, EU Ltd. (Novartis Gene Therapies) a new unit dedicated to developing innovative AAV-based gene therapies. While the name of the organisation changes, the commitment to patients and their families remains steadfast. Novartis Gene Therapies will continue to focus on the research, […]

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  2. Zolgensma®, the first gene therapy to treat SMA, receives conditional marketing authorisation in the European Union

    On 19th May, the European Commission issued a conditional marketing authorisation for the breakthrough gene therapy drug onasemnogene abeparvovec (Zolgensma®) for treating spinal muscular atrophy. This is the second drug developed to treat this severe neuromuscular disorder to receive approval in the European Union. Onasemnogene abeparvovec, marketed as Zolgensma®, is authorised for use in treating […]

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  3. European Medicines Agency grants positive recommendation for conditional marketing authorisation of gene therapy product onasemnogene abeparvovec (Zolgensma®)

    On 27th March, the European Medicines Agency recommended a conditional approval of the breakthrough gene therapy drug onasemnogene abeparvovec (Zolgensma®) for treating spinal muscular atrophy. SMA Europe is thrilled that a second therapy that addresses the causes of this life-limiting disease has moved closer to patients. It is a culmination of efforts that have been […]

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  4. COVID-19

    Dear community, This strain of coronavirus is new, which means the knowledge on how it will affect people living with SMA is still limited. However, this is a rapidly changing field so advice will be updated as more is learned. We have built a repository of information, where this information will be uploaded. Leading experts […]

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