Substrate-reducing ASOs show promise in Pompe mouse model

Substrate-reducing ASOs show promise in Pompe mouse model

A new type of substrate reduction therapy composed of antisense oligonucleotides, or ASOs, effectively reduces glycogen accumulation in the skeletal muscles, or those attached to bones, of a Pompe disease mouse model, a study found. When combined with standard enzyme replacement therapy (ERT), which showed limited efficacy in skeletal muscles, motor performance improved further than with the ASO alone. “These findings highlight the potential of …