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. Evrysdi improves motor function in pre-symptomatic babies after one year and its safety profile in previously treated people with SMA is confirmed

    In response to our request, Roche has shared new interim data from two studies evaluating risdiplam in people with SMA, who have previously been treated with other SMA-targeting therapies (JEWELFISH) and pre-symptomatic babies (RAINBOWFISH). The findings were presented at the Cure SMA 2021 Virtual SMA Research & Clinical Care Meeting, which is taking place from […]

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  2. Zolgensma Summary of Product Characteristics Updated

    The Summary of Product Characteristics (SmPC) for Zolgensma has been updated. The SmPC is a document describing the properties and the officially approved conditions of use of a medicine. Summaries of product characteristics form the basis of information for healthcare professionals on how to use the medicine safely and effectively. Novartis Gene Therapies is committed […]

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  3. European Commission approves Evrysdi for the treatment of 5qSMA

    Following our request for regular updates, Roche is delighted to inform us that today the European Commission (EC) approved Evrysdi™ (risdiplam), the first and only at home treatment for SMA, with proven efficacy in adults, children and babies two months and older. Risdiplam was reviewed under the European Medicine Agency’s (EMA) accelerated assessment programme, intended […]

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  4. Community update on access to Spinraza in Europe

    In response to our request, Biogen has provided us with an update on access to SPINRAZA™ (nusinersen). There are now 29 European countries that have access to nusinersen via regular reimbursement. Since the last report, an updated situation can be found for Switzerland. As you can see from the table, there is a range of […]

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