Below we list all of the treatments which have recently been the subject of clinical trials and are currently available for use by adults in the UK.
Raxone (idebenone) – produced by Santhera pharmaceuticals
Raxone tablets are available in the UK through the Early Access to Medicines Scheme (EAMS).Raxone has been approved for use within the EAMS programme to slow the rate of decline of breathing ability in patients with DMD from the age of 10 years upwards who are not taking glucocorticoids (also known as ‘steroids’).
During the EAMS period Santhera will provide Raxone free of charge to the NHS. The scheme will close once a formal product licence has been given for the treatment. Once the scheme closes, Santhera will continue to supply the medicine for people who are already enrolled in the EAMS for as long as their doctor considers it to be beneficial, up to the point of a positive NHS funding policy.
Treatment will only be provided in specialist centres that are experienced in managing Duchenne and have enrolled in the scheme. There is a requirement for careful monitoring to ensure the safety of patients during the EAMS.
This medicine may not be suitable for everybody and people with DMD need to discuss their own condition with a specialist doctor who knows them to assess if this
treatment is appropriate.