Myotonic Dystrophy Expertise Center

Introduction

Basic and clinical researchers at Radboud university medical center (Radboudumc) and Maastricht University Medical Center (MUMC+) have joined forces in a translational research program on Myotonic Dystrophy (DM). The research program is closely connected to the NFU/VWS accredited “expertisecentrum DM” for specialist care, treatment and research and participates in the Academic Alliance MUMC-Radboudumc, the Netherlands Neuromuscular Center (NL-NMD) and the European Reference Network for rare neuromuscular diseases (EURO-NMD).

Our mission

We are committed to transforming care and cure for people living with myotonic dystrophy (DM). We design and implement personalized, evidence-based interventions that directly improve patient outcomes.

Why partner with us?

With multiple phase III therapies emerging for DM, now is the time to accelerate development and implementation. The Radboudumc/MUMC+ Expertise Center is a leading global hub for DM research, covering the full spectrum from basic science to clinical trials and real-world care.

What we offer:

  • MYODRAFT registry with >800 DM1 patients (adult & pediatric) and up to 7 years of longitudinal follow-up, combining patient-reported and clinician-validated data
  • A biobank with blood, DNA, serum,  muscle and skin biopsies
  • Biomarker identification and validation with state-of-the-art platforms
  • Extensive cell line platforms for drug screening, including primary and iPSC-derived cell lines
  • DM1-specific outcome measure development
  • A hub for ongoing and future clinical trials, with rapid recruitment and well equipped trial unit

Our integrated infrastructure spanning data, models, biomarkers, and clinical expertise, supports efficient therapy development, validation, and implementation. We actively collaborate with industry to translate innovation into meaningful impact for people living with DM.

Contact us for questions or collaboration

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