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. The liver is a therapeutic target in SMA.

    Professor Tom Gillingwater and Professor Simon Parson, at Edinburgh & Aberdeen Universities respectively, have found that the liver is a therapeutic target in SMA. The results of this investigation have been published in the Nature journal, Scientific Reports. This study was partly funded by The SMA Trust and SMA Europe, amongst others. Decreased levels of […]


  2. A role for mitochondria in SMA

    A team at Stanley Manne Children’s Research Institute at Ann & Robert H. Lurie Children’s Hospital of Chicago discovers a role for mitochondria in SMA. They identified functional and structural defects to the cells’ power houses, called mitochondria, which contribute to the motor neuron’s degeneration. These defects occur before the onset of SMA symptoms. Although the […]


  3. EMA to hold stakeholder workshop for SMA

    Following a request by SMA Europe, the European Medicines Agency (EMA) will host a one-day workshop on spinal muscular atrophy (SMA) on 11th November this year, at its headquarters in London to allow SMA stakeholders to have an open forum discussion on the current challenges that face therapy development for SMA. This initiative is very important as […]