Hundreds of children born with a degenerative muscle-wasting disease are to be offered a new drug on the NHS that can slow the deadly condition’s progression.
Duchenne muscular dystrophy (DMD) is an incurable genetic condition which causes the muscles to slowly stop working. Most patients will not live past 30.
The new treatment, vamorolone, has been seen in trials to buy time for DMD patients and allow them to stand and walk for longer.
Earlier this year the NHS spending watchdog, the National Institute for Health and Care Excellence (NICE), said it would not fund vamorolone, which costs about £4,500 for three months’ worth of treatment.
But it has now reversed its decision after a deal was struck with manufacturer Santhera Pharmaceuticals to provide the drug to the NHS at an undisclosed lower price.
Emily Reuben, chief executive of Duchenne UK, and Alex Johnson OBE, chief executive of Joining Jack, both have sons with the condition and have long called on the NHS to invest more into pioneering DMD treatments.
In a joint statement they said: ‘We were given very little hope when our sons were diagnosed with DMD. We are very proud that a treatment that we have supported for so long will now be available on the NHS.’
DMD affects about 20 babies in the UK every year – almost all of them boys as the gene that triggers the condition is linked to the male chromosome.
Duchenne muscular dystrophy (DMD) is an incurable genetic condition which causes the muscles to slowly stop working. Most patients will not live past 30 (Stock image)
Those with DMD lack the dystrophin protein, which leads to muscle fibres slowly breaking down and being replaced by fatty tissue, gradually weakening muscles.
Steroids are used to treat DMD, but have limited success and unpleasant side effects, such as thinning bones, which raises the risk of life-threatening fractures.
Vamorolone comes as a liquid consumed once daily and works in a similar way to steroids by stopping the production of inflammatory cells that damage the muscle cells of patients. However, it also avoids triggering the side effects linked to long-term steroid use.
