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. 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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  2. 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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  3. 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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