Treatment for Duchenne is currently largely limited to glucocorticoids that have been shown to prolong ambulation and also help to prevent scoliosis. More satisfactory treatments are urgently needed. Efforts are focused on identifying drugs or biological agents that have the potential to maintain long term muscle function alongside an acceptable side effect profile.
Multiple treatment approaches have emerged. Some address the underlying cause of Duchenne which is a lack of dystrophin, and others look at fixing the symptoms caused by the effects of lack of dystrophin in the muscle.
Developing a new drug is a costly and complicated process that can take many years. This can be very difficult for families to manage, for whom every minute counts. However the landscape has changed beyond recognition from 10 years ago. Some drugs are now being tested on patients with Duchenne Muscular Dystrophy and many others are about to start clinical trials.
We’ve summarised the different approaches below. You can also read more about how clinical trials work here and what things you might like to consider if you do want your child to take part in research.
An exciting new gene-based therapy on the horizonRead more
Some promising steroid alternatives may not have the side-effects of traditional treatments.Read more
Exon skipping drugs are small pieces of genetic material that can help patch over missing parts of the gene.Read more
Reducing myostatin may help to make the muscles of those with DMD more healthyRead more
Already existing drugs developed for other conditions could be helpful to those with DMDRead more
Nutraceuticals are products derived from food sources that provide extra health benefits, in addition to the basic nutritional value found in the food. Many nutraceuticals are thought to be beneficial to DMD.Read more
CRISPR /Cas 9 is an exciting genetic engineering technique.Read more
Stem cells are the building blocks of the body. New research tries to manipulate them to help DuchenneRead more
Around 90% of those with DMD have associated heart problems. New research aims to target thisRead more
Increasing the levels of a protein called utrophin may help those with DMD regardless of their mutation typeRead more
Research aims to discover how to treat muscle fibre loss, where muscles are replaced by scar tissue.Read more