Where would we be without funding from Muscular Dystrophy UK? The basic answer is we wouldn’t be anywhere.
Professor Doug Turnbull, Professor of Neurology at the University of Newcastle.
There are currently no treatments for most of the muscle-wasting conditions we support. Research generates the knowledge that is essential for understanding the cause of these conditions. This knowledge can then be used in finding effective treatments.
Since 1959 we have invested more than £55 million in high quality research into muscle-wasting conditions. This has been possible only through the generosity and dedication of our supporters.
We use a robust international peer-review system, and have laid the foundations for the first potential treatments for some muscle-wasting conditions.
We have contributed to crucial scientific breakthroughs:
- laying the foundations for the promising technology of ‘exon skipping’ which is currently being tested in clinical trials for boys with Duchenne muscular dystrophy
- funding work that has led to a scientific breakthrough in finding a treatment for mitochondrial myopathy, now close to clinical trial.
We are also pressing to ensure early participation for UK patients in clinical trials. We fund clinical trial co-ordinators and the national neuromuscular database to ensure vital patient information is available to clinicians and researchers.
We won’t stop until there are treatments and cures for all muscle-wasting conditions. We cannot do this without your support – please help us now.
The technique corrected the exon 50 mutation in a DMD mouse model
Latest details on three new gene therapy trials
Sarepta publishes data from its small phase 2 studies
To develop gene therapies for LGMD
24 January 2018
27 January 2018
2 February 2018