News Archive - SMA Europe

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. Scholar Rock Announces Positive Opinion by the EMA on Orphan Drug Designation for SRK-015 for the Treatment of SMA

    Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the European Medicines Agency’s (EMA’s) Committee for Orphan Medicinal Products (COMP) has adopted a positive opinion recommending SRK-015 for designation as an orphan medicinal product for the treatment of SMA. […]

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  2. Novartis announces FDA filing acceptance and Priority Review of AVXS-101

    Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA®(onasemnogene abeparvovec-xxxx), an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. ZOLGENSMA is designed to address the genetic root cause of SMA Type 1, a […]

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  3. Scholar Rock Reports Preclinical Data on the Therapeutic Benefit of SRK-015 in Models of SMA

    Scholar Rock, a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced the publication, “Specific Inhibition of Myostatin Activation is Beneficial in Mouse Models of SMA Therapy” in the peer-reviewed journal Human Molecular Genetics. The publication details preclinical studies demonstrating that a highly specific […]

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  4. Roche provides community update on FIREFISH & SUNFISH studies of risdiplam

    Roche has provided us with the following statement on the FIREFISH and SUNFISH studies of risdiplam: Dear SMA community, We are happy to share an update on our FIREFISH and SUNFISH studies of risdiplam. We would also like to acknowledge the tremendous support and partnership of SMA Patient Groups around the world and to thank […]

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