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.
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.
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.
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.