According to Medical Xpress, there may be a new treatment option for patients with Duchenne muscular dystrophy (DMD). Traditionally, DMD is treated with glucocorticoids. However, these often require high doses and result in serious side effects. Now, a new study suggests that vamorolone may improve patient outcomes while reducing adverse reactions. See the full findings published in PLOS Medicine.
For patients with DMD, a steroid regimen (prednisone, deflazacort) is the traditional standard of care. However, long-term steroid use may cause problems with growth, bone health, and bone development. But recent data suggests that vamorolone, in conjunction with steroids, reduces the negative impacts while also retaining the steroidal efficacy.
According to ReveraGen BioPharma, vamorolone is:
an investigational drug that was developed as a dissociative steroid. This means that its chemical profile is such that it may be shown to chemically separate the aspects of efficacy (clinical benefit) from safety concerns (side effect profiles).
The therapy is anti-inflammatory as well. The goal of the drug is to retain the benefits of steroid therapy (mobility, quality of life, overall survival) while reducing the negative symptoms (poor development, bone health problems). Researchers are particularly interested in the efficacy of vamorolone for young male patients, whose muscle weakness becomes more progressive following the age of six.
In the six month trial, researchers enrolled 46 participants. Following the trial, 100% of patients chose to continue vamorolone use. The therapy increased muscle strength and muscle function.
Duchenne Muscular Dystrophy (DMD)
Overall, there are nine forms of muscular dystrophy. Patients with Duchenne muscular dystrophy lack the ability to create dystrophin. According to the Genetics Home Reference, dystrophin is:
part of a group of proteins that work together to strengthen muscle fibers and protect them from injury as muscles contract and relax. The dystrophin complex acts as an anchor, connecting each muscle cell’s structural framework (cytoskeleton) with the lattice of proteins and other molecules outside the cell (extracellular matrix).
As a result, patients experience skeletal and heart muscle weakness. DMD is much more common in males (1 in 3500 births) as opposed to females (1 in 50 million births). This is because the mutation which causes DMD is in the X chromosome. Females need two mutated copies to inherit DMD, while males need only one.
Symptom onset occurs before age 6. These include:
- Developmental delays
- Learning disabilities
- Difficulty walking, climbing, or moving positions
- Progressive muscle weakness
- Poor motor function
- Heart disease (progression)
- Respiratory failure (progression)
Learn more about DMD.