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

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.