Biogen presented data at the American Academy of Neurology 2019 conference on a potential biomarker for SMA. Biomarkers provide insight on disease progression.

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.