Experimentele therapie bij duchenne-spierdystrofie

Duchenne muscular dystrophy is an X-chromosomal inherited muscle disease.

Absence of the protein dystrophin results in a decreased stability of the muscle membranes. This leads to progressive muscle weakness starting at childhood.

Although corticosteroids can be used to slow down the course of the disease, no treatment for Duchenne muscular dystrophy is available yet.

Several lines of research are exploring possible treatment options. These include mutation specific medication, for example antisense oligonucleotides (exon skipping), as well as therapies that could possibly be used for other muscle diseases as well, such as the inhibition of muscle degeneration and stimulation of muscle regeneration.

In this article an overview of the therapeutic developments for Duchenne muscular dystrophy is presented.