The researchers said that the muscle damage caused by Spinal muscular atrophy (SMA) in babies can be reversed by using a class of drugs known as HDAC inhibitors. SMA is a motor neuron disease that affects around one in 6,000 babies with more than half of those suffering from the most severe form of the disease dying before the age of two years.
Researchers looking into the disease found that SMA not only led to muscle damage but also caused unhealthy muscles due to reduced blood supply to the muscle tissues. The researchers tested the effect of HDAC inhibitors on a group of mice affected with SMA and found that the drugs targeted the genetic mutation and increased the levels of SMN protein in the affected muscles.
"Spinal muscular atrophy is the most common genetic cause of death in children. By showing the important role that muscles play in this disease, we can now focus our efforts on trying to block the disease in all affected tissues of the body", lead author Professor Tom Gillingwater said. The study has been published in the journal Human Molecular Genetics and Neuromuscular Disorders.