SMA Newsroom

Clinical trialsMay 9, 2019

Data published in Neurology suggest long-term benefit of Spinraza™ treatment in later-onset SMA, including SMA type III

Biogen has announced that data from a study on the safety and tolerability of Spinraza™ in individuals with later-onset SMA had been published in the peer-reviewed journal Neurology.

Clinical trialsFeb 27, 2019

Scholar Rock Announced Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans

Scholar Rock announced positive interim results from the ongoing Phase 1 clinical trial of its potential muscle-targeted SMA therapy, SRK-015 in healthy adult volunteers.

OtherFeb 25, 2019

SMA highlighted at Eurordis’ multi-stakeholder symposium to improve patient access to rare disease therapies

Over 250 rare disease stakeholders gathered in Brussels on 13th & 14th February to develop and endorse Eurordis’ new proposed framework to sustainably improve patient access to rare disease therapies.

Clinical trialsDec 5, 2018

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.