The Spinal Muscular Atrophy (SMA) Foundation has announced
the launch of a pilot study of Biomarkers for Spinal Muscular Atrophy
(BforSMA). The Foundation is a nonprofit organization dedicated to accelerating
the development of a treatment for SMA.
The goal of this study is to identify a potential biomarker or panel of biomarkers that can be used as objective measures of SMA disease severity. The identification of such biomarkers may help to assess drug efficacy and shorten the duration of clinical trials.
To ensure wide outreach and quick completion of the study, 18 clinical investigators have been recruited to participate in BforSMA. The first patient is expected to enroll in October. The study aims to enroll 100 children with SMA, making this one of the largest research efforts in SMA history. The Foundation has also recruited the New England Research Institutes (NERI), BG Medicine and Expression Analysis as research partners in this project.
Cynthia Joyce, Executive Director for the SMA Foundation, added, "This pilot project has the potential to be a landmark study for therapeutics development - rapid completion will help make future clinical trials more efficient and more informative.