Annemieke Aartsma-Rus has made an important contribution to research in Duchenne muscular dystrophy. Patients with this serious hereditary muscle disease lack the protein dystrophin, because the gene code for dystrophin is unreadable. Thanks in part to the fundamental pioneering work of Aartsma-Rus, an exon skipping therapy is now available in the USA and Japan that can make the genetic code readable again. This can slow down disease progression for Duchenne patients.
