The results of an interim trial, with 46 participants, suggest that the synthetic steroid Vamorolone may offer a better alternative to long-term treatments than current steroids for young Duchenne Muscular Dystrophy (DMD) patients.
Conducted at Duke University Medical Centre, this year-long global DMD trial reveals that Vamorolone could replace treatments with long-term high-dose glucocorticoids, which can increase health risks within patients. For example, the reduction of development of growth plates in young sufferers and hinders the lengthening of their bones.
Currently, steroid therapy is the only available treatment for DMD as it helps to slow the effects of the irreversible, genetic disease and has extended people’s lives and mobility.
Unfortunately, many DMD patients only live into their 30s, at which they commonly experience respiratory and heart failure.
A clinical investigator in the trial and co-director of the Duke Children’s Neuromuscular Program, neurologist Dr Edward Smith, states “One of our biggest concerns about high-dose steroid treatment in these patients is the effect on linear growth and bone development.
“So far, based on the interim results from this trial, we may be seeing a much less negative impact on bone health among patients using Vamorolone.”
The progressive DMD is caused by X chromosome mutation that results in hereditary incapability to produce dystrophin – a protein that preserves skeletal and heart muscles from damage caused by natural contraction and relaxation.
There are more than 60 rare and very rare progressive muscle-weakening and wasting conditions. Muscle-wasting conditions are very rare, affecting just over one in every 1,000 people and affecting 70,000 people in the UK.
We are experienced in providing highly tailored care to enable individuals with muscle-wasting conditions to live their lives to the full.
To find out more about our Muscular Dystrophy services, contact us today.