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 (chaired by Prof. Prof. dr. Silvère van der Maarel) 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.

ballooningThis website is created for parents and family of DMD patients. On this website we try to explain the exon skip therapy with non-scientific language and simple images. If something on this website is unclear, or if you want to use our information for a presentation or public speech, do not hesitate to contact us by using the contact form.

Recently a new video has been created to show a “bit more behind the scene footage” of our work here in Leiden and tells a bit more about exon skipping and the DMD disease.

Please have a look!

About Duchenne Muscular Dystrophy (DMD)

home plaatje
Here you will find more background information about the disease.

 Our publications about the disease and the research

Publications Here you will find all the scientific publications about Duchenne.

About the Exon skipping technique

AON treatment
Here you will find more background information about the Exon skipping technology

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