The research aims to:
Elucidate the genetic and molecular basis of a group of diseases called congenital muscular dystrophies.
Regulate splicing in Duchenne muscular dystrophy and spinal muscular atrophy.
Characterise muscle stem cells for possible future therapeutic applications.
You can find out more information about our current research projects on the Dubowitz Neuromuscular Centre research page on the UCL Institue of Child Health website.
MRC Neuromuscular Translational Research Centre and TREAT-NMD
The Dubowitz Neuromuscular Centre is one of the members of the MRC Neuromuscular Translational Research Centre. For more information visit the MRC Centre for Neuromuscular Diseases website.
The ICH is part of TREAT-NMD, which is a network that brings together people with neuromuscular diseases and specialists (scientists, healthcare professionals and pharmaceutical companies) working on treatments for these conditions.
Linking 22 institutions and over 300 doctors, researchers and other professionals throughout 11 European countries, the network is enabling experts to work together to share good practice and improve global standards of care.
The TREAT-NMD network received funding from the European Union to help speed up the research that is being done into neuromuscular diseases, so that the most promising new therapies can move more quickly from being an idea in a laboratory to a real treatment for patients.
Recent advances in cutting-edge therapies mean that clinical trials are now a reality. But such advances are being delayed because of the fragmentation that currently exists in research and healthcare systems across the globe.
Without validated outcome measures, consensus on standards of care and diagnosis, or uniform patient databases, setting up international trials of the most promising therapies is an uphill struggle.
TREAT-NMD addresses all these areas of concern, with working groups of partners devoted to each of the major issues.
For more information, visit the TREAT-NMD website.