Can the drug acipimox relieve muscle symptoms in patients with mitochondrial myopathy?
Mitochondrial Diseases / Posted 11 months ago
Mitochondria are tiny cell structures that play a critical role in the production of energy by the cell. When these tiny energy producing cell structures malfunction, it may result in chronic illnesses known as mitochondrial diseases. It is estimated that mitochondrial disease can affect up to 15,000 adults (and a similar numbers of children) in the UK. Mitochondrial diseases are rare, inherited conditions where muscle symptoms such as muscle weakness, tiredness and pain are extremely common and often draining. The study has been designed with the help of patients and carers who identified muscle symptoms to be the most important target for any new drug treatments. We plan to test a drug (acipimox) which has previously been used to treat high cholesterol and improve diabetic control. The drug has also been shown to boost production of cell energy by muscle cells and it is this role that we wish to test in patients with mitochondrial disease to see if we can to ease the unbearable muscle symptoms which are suffered.