News | SMA Europe - Part 2

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. 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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  2. Alliance strives to bridge the newborn screening gap to include SMA!

    It’s time for Europe to bridge the newborn screening gap and include spinal muscular atrophy!   The European Alliance for Newborn Screening in Spinal Muscular Atrophy calls upon the government of Europe to include a test for spinal muscular atrophy in national newborn screening programmes.   Worldwide newborn screening projects and programmes for SMA clearly […]

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  3. Financial boost to develop promising regenerative treatments for neuromuscular diseases

    The Spinal Muscular Atrophy (SMA) Foundation and PTC Therapeutics have entered into a new collaboration focused on regenerative medicine to further advance scientific research in SMA and other neuromuscular disorders. Recent FDA approvals of several disease modifying therapies for SMA have made regenerative medicine approaches the next frontier in drug discovery and development. These approved […]

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