Hundreds of children born with a degenerative, muscle-wasting disease will be offered a new drug on the NHS that can slow the progression of the deadly disease.
Duchenne muscular dystrophy (DMD) is an incurable genetic condition that causes muscles to slowly stop working. Most patients will not live longer than 30 years.
The new treatment, vamorolone, has been seen in trials to buy DMD patients time and allow them to stand and walk for longer.
Earlier this year, the NHS’s spending watchdog, the National Institute for Health and Care Excellence (NICE), said it would not fund vamorolone, which costs around £4,500 for a treatment of three months.
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, have children with the condition and have long called on the NHS to invest more in pioneering treatments for DMD.
In a joint statement they said: ‘We were given very little hope when our children were diagnosed with DMD. “We are very proud that a treatment we have supported for so long is now available on the NHS.”
DMD affects around 20 babies in the UK each year, almost all boys, as the gene that triggers the condition is linked to the male chromosome.
Duchenne muscular dystrophy (DMD) is an incurable genetic condition that causes muscles to slowly stop working. Most patients will not live longer than 30 years (file image)
People with DMD lack the protein dystrophin, which causes muscle fibers to slowly break down and be replaced by fatty tissue, gradually weakening the muscles.
Steroids are used to treat DMD, but they have limited success and unpleasant side effects, such as thinning bones, which increases the risk of life-threatening fractures.
Vamorolone comes in liquid form consumed once a day and works similarly to steroids by stopping the production of inflammatory cells that damage patients’ muscle cells. However, it also avoids triggering the side effects related to long-term steroid use.