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. EMA to hold stakeholder workshop for SMA

    EMA SMAFollowing 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 EMA plays a crucial role in the innovation and availability of medicines within the European Union and beyond. The Agency aims to improve the science and process of medicines regulation, in order to foster the development of medicines for unmet medical needs, such as SMA and to optimise their surveillance and management on the market. To do this, the agency fosters scientific excellence by ensuring it is aware of the latest scientific knowledge and methodologies by targeting engagement with stakeholders in a range of areas. We are therefore delighted that the agency has recognised the high need for SMA and agreed to host a workshop, which has now been advertised on their website.

    This workshop is being co-organised with TREAT NMD and EMA and will bring together key stakeholders to discuss, help and advance the development of therapies for the treatment of SMA. Topics for discussion will include an overview of the disease, the pharmacology of the molecules under investigation, natural history data, clinical outcome measures and biomarkers.

    We look forward to report on the outcome in due course.