Potential therapies and biomarkers for LAMA2-CMD. Does the microRNA hype deliver?
Laminin α2 chain-deficient muscular dystrophy, or LAMA2-CMD, is a very severe disease caused by mutations in the LAMA2 gene. Skeletal muscle is the most affected tissue, with patients presenting symptoms such as hypotonia at birth, joint contractures and progressive muscle wasting. Changes in the central nervous system include white matter abnormalities, delayed motor milestones and compromised ac