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. AveXis provides community update on plans for AVXS-101 in Europe

    AveXis has provided SMA Europe with a community update on plans for AVXS-101 in Europe. AVXS-101 is a gene therapy product for SMA. STR1VE EU OVERVIEW: STR1VE EU is expected to start enrolling patients in the first half of 2018 in patients with SMA Type 1, studied at multiple centres across the European Union. ADMINISTRATION: […]

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  2. AVEXIS announced Phase 1 clinical trial of AVXS-101 for SMA Type 2

    Avexis has been granted authorisation to start Phase 1 clinical trial of AVXS-101 for patients with SMA Type 2 by the U.S. Food and Drug Administration (FDA), based on review of data submitted. The company plans to initiate this trial immediately. The open-label, dose-comparison, multi-center Phase 1 trial named STRONG, is designed to evaluate the […]

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  3. The patient voice at EMA

    The European Medicines Agency (EMA) is the European Union agency responsible for the evaluation and supervision of medicines. Hear the voices of EMA’s stakeholders – Patients and healthcare professionals representatives explain the value of working with the Agency for better decision-making and how important it is to ensure your voice is added to the regulatory […]

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  4. Roche provides community statement regarding SUNFISH

    Roche provides community statement regarding SUNFISH, a clinical trial of RG7916 for SMA Types 2/3. An interim analysis was recently presented at the International Conference of the World Muscle Society in France. In this analysis, all participants (51 people) had received RG7916 for 28 days or longer. SMN protein increased by up to two and […]

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  5. AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1

    The U.S. Food and Drug Administration (FDA) has notified AveXis that, based on review of data submitted, including the potency assay, it may initiate its planned pivotal trial of AVXS-101 for patients with SMA Type 1 using the intravenous (IV) formulation produced by the company’s Good Manufacturing Practice (GMP) commercial manufacturing process. The company plans […]

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