SMA Newsroom

ResearchMay 9, 2019

Potential biomarker for SMA identified

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

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.