Welcome to the information website for parents of Duchenne patients

DMD Genetic Therapy group Leiden

The primary mission of the DMD Genetic Therapy Group at the Department of Human Genetics of the Leiden University Medical Center  is the development of a genetic therapy for Duchenne muscular dystrophy (DMD). The project started in 1998 and is currently supervised by Prof. Dr. Annemieke Aartsma-Rus, after Dr. Judith C.T. van Deutekom left in February 2007 to work as Head of research for the biotech company Prosensa B.V. (Leiden, the Netherlands). In a joint effort the DMD Genetic Therapy Group and BioMarin (previously Prosensa) are developing the exon skip therapy for Duchenne.

This website is made for parents and family of DMD patients. On this website we try to explain the exon skipping theory with non-scientific terms and simple figures. If something is unreadable or if you want more information for a presentation or talk, please contact us. We would like to inform you more about Duchenne. Besides, in this way you help us to improve the website.

The video below has been created to show a bit more about Duchenne, the exon skipping technique and our work. In case you would like to read more scientific information about this you can visit the website of the DMD Genetic Therapy group.

Please have a look!

Information Duchenne

Background information about Duchenne muscular dystrophy

Exon skipping

Information about the exon skipping technique we use

Research publications

All scientific publications from the DMD Genetic Therapy group