Project partners

Leiden University

Leiden University Medical Centre

Leiden University Medical Centre (LUMC) has an international reputation for  bridging fundamental research and patient care. The LUMC trains researchers and clinical specialists in many areas. They have a long standing interest in NMDs and Duchenne muscular dystrophy in particular and were involved in the identification of the DMD gene. LUMC has a dedicated clinical team for neuromuscular disorders and peerrforms DNA diagnostics for NMD patients throughout the Netherlands. In addition, they have been one of the pioneers in genomic, transcriptomic and proteomic biomarker identification for NMDs.

LUMC's website

Contact: Peter 't Hoen
Email Peter

Department of Human Genetics DMD Genetic Therapy Group
Post-Zone S04-034
LUMC - Human Genetics
Postbus 9600
2300 RC Leiden
T: +
31 71 526 9421


Leiden University and BIO-NMD

LUMC is the leader for work package 3 - Proteomic biomarkers discovery by studies on patient cells, muscle tissues and body fluids, and work package 4 - Exploratory biomarkers validation in humans. This is based on the partner's previous expertise with biomarker identification in NMD mouse models. LUMC is also involved as a partner in other work packages.


Peter Bram’t Hoen

Peter-Bram’t Hoen is an assistant professor at the Department of Human Genetics. He has set up high-throughput RNA and protein profiling studies for muscle diseases, established a muscle gene expression databases containing data from all studies published and supervises research on the identification of biomarkers that correlate with disease severity in several mouse models for muscular dystrophies. These biomarker profiles can be used to assess therapeutic effects after (AON) treatment.


Annemieke Aartsma-Rus

Annemieke Aartsma-Rus is an associate professor at the Department of Human Genetics and leader of the DMD genetic therapy group. She is the scientific representative of the LUMC in the TREAT-NMD governing board. During her PhD research she was involved in the development of therapeutic antisense-mediated exon skipping for DMD. She successfully defended her thesis titled “Development of an antisense-mediated exon skipping therapy for Duchenne muscular Dystrophy – Making sense out of nonsense” in February 2005 and now continues to work on the further optimization of the exon skipping therapy.



FP7 & EU flag