Ataluren and Raxone are used as treatments for DMD in the UK. Eteplirsen is approved in the USA. We are working hard to fund research to find effective treatments and a cure for everyone diagnosed with DMD.

Ataluren (also known as Translarna)

Ataluren is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged five years and older.

Ataluren is a treatment for patients who have a particular type of defect in the DMD gene called a nonsense mutation.

A nonsense mutation creates a premature stop signal in the gene that causes the creation of the dystrophin protein in the cell to stop prematurely. This results in a truncated protein that cannot function normally and that is subsequently destroyed by the cell. Approximately 13 percent of DMD cases are caused by this type of mutation.

Ataluren forces the cell to ignore this abnormal premature stop signal, enabling the production of the full-length, functional protein. Thus, Ataluren works as a “protein restoration therapy”, which means that it aims to facilitate the production of a functional protein in patients who cannot produce it normally.

Find out more here.

Raxone (also known as Idebenone)

Raxone is NOT an approved treatment. However it is available to some patients on the Early Access to Medicines Scheme 

Raxone is available in the UK under the Early Access to Medicines Scheme, Santhera are providing the drug to the NHS free of charge for patients who qualify for it.

Raxone is a drug produced by Santhera Pharmaceuticals. The active substance in Raxone is called idebenone, which is believed to improve energy production in muscle cells. Raxone is believed to help slow down the decline in respiratory function for DMD Patients by slowing the rate at which the respiratory muscles weaken. 

Find out more here.

Treatments approved in the USA

Please note this is not approved in the UK.

Eteplirsen (brand name Exondys 51)

Eteplirsen is a drug produced by Sarepta Therapeutics to treat patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. Eteplirsen only targets specific mutations so can treat about 14% of patients with DMD.

This indication is approved under accelerated approval based on an increase in dystrophin in skeletal muscle observed in some patients treated with EXONDYS 51.

Find out more here.