Enzyme Replacement Therapy May Stabilize Pompe Disease Gene Expression
Transcriptomic gene analysis of skeletal muscle biopsies from patients with late-onset Pompe disease (LOPD) suggests that enzyme replacement therapy attenuates the expression of dysregulated genes involved in lysosomal function, according to a recently published study in Molecular Genetics and Metabolism. Although enzyme replacement therapy has significantly improved the survival and quality of life of many patients…