NIHR awards £450,000 for rare neuromuscular diseases

The award was made to the rare neuromuscular theme which is led by Professor Michael Hanna, Director of UCL's Institute of Neurology, where research projects into Duchenne Muscular Dystrophy (DMD) and inclusion body myositis (IBM) have been funded.

Professor Francesco Muntoni, supported by the Great Ormond Street BRC, will study groups of patients with DMD and measure their particular genetic defects more precisely. 

Professor Hanna said: "The DMD programme is going to collect large cohorts nationally through the BRC infrastructure to study their natural history and their genetics accurately. We aim to understand the variation in disease severity and assess variations in treatment response in therapeutic trials."

This will be a valuable step in the translational pathway for DMD patients.

The second study will explore IBM, an inflammatory muscle disease depicted by progressive weakness and wasting of both distal and proximal muscles. Professor Hanna explained how BRC work on stratification of patients based on muscle biopsy appearances, clinical assessment and MRI evaluation will be valuable in designing and involving patients in clinical trials.

Based on the BRC's strong links with industry it is highly likely trials of two new agents will be able to start in the next couple of years.