According to a story from Newswise, trial results released in the journal PLOS Medicine suggest that the drug vamorolone could provide an enhancement in the effectiveness of corticosteroid therapy in patients living with Duchenne muscular dystrophy, a rare genetic disorder. More interestingly, the study authors also suggest that the medicine could serve as an alternative to corticosteroid therapy and could also be effective in other diseases in which corticosteroids are commonly used.
About Duchenne Muscular Dystrophy (DMD)
Duchenne muscular dystrophy is a neuromuscular disease, and it is one of the more severe types of muscular dystrophy. It is characterized by progressive muscle weakness that usually begins around age four and worsens quickly. As an X-linked genetic disease, males are mostly affected, with females only occasionally displaying mild symptoms. The disease is caused by mutations of the dystrophin gene. Symptoms of Duchenne muscular dystrophy include falling, abnormal walking posture, eventual loss of walking ability, muscle fiber deformities, intellectual disability (not in all cases), enlargement of the tongue and calf muscles, skeletal deformities, muscle atrophy, heart abnormalities, and difficulty with breathing. Treatment includes a variety of medications and therapies that can help alleviate symptoms and slow disease progression. Lifespan is usually into the thirties with good care. Better treatments for this disease are urgently needed. To learn more about Duchenne muscular dystrophy, click here.
Corticosteroids have been a component of treatment for this rare disease for a while as they help to bring down inflammation affecting the muscles and have been shown to improve overall quality of life. With that being said, a high dosage is often needed, and long term use can lead to safety issues and severe side effects.
Reduced Side Effects
The researchers hope that treatment with vamorolone can be used to bring down inflammation, with the dose increasing as the patient ages. This isn’t possible with corticosteroids. Ideally, this approach would be able to slow down the progression of the disorder. The trial included patients between ages four to seven who hadn’t been treated with steroids before. These patients were given vamorolone for 18 months and had their motor functions monitored for six months afterward.
The scientists saw improvements in climbing stairs, running, and walking speed. Meanwhile, the potential side effects of steroids were not present. These findings suggest that this medicine could allow for a real increase in quality of life for Duchenne muscular dystrophy patients.
Check out the original study here.