The Griffins of Harrisburg, North Carolina enrolled their son in the AGAMREE clinical trial in 2016. Their son was the first participant in the trial studying the investigational drug vamorolone for the treatment of Duchenne muscular dystrophy (DMD). He has received positive results and has remained on the treatment along with other families at Duke University’s MDA Cancer Care Center. The Griffins will readily acknowledge that their son has been relatively free of the typical adverse reactions to steroids.
Other boys saw an improvement in bone health leading to avoidance of painful fractures. Common side effects are cushingoid features (Cushing syndrome), psychiatric disorders, vomiting, weight gain and vitamin D deficiency. The attending physicians anticipate a delayed loss of ambulation.
About Duchenne Muscular Dystrophy (DMD)
DMD is a progressive muscle disease which is caused by the lack of dystrophin that normally stabilizes muscle cells. The absence of dystrophin causes muscle weakness and muscle deterioration. By the age of eight or nine, especially in the absence of physical therapy, the child may need leg braces. DMD usually affects young boys. Estimates are that there are approximately six cases for every 100,000 individuals. Eventually the disease becomes fatal as DMD impacts heart and lung function. Currently, there is no cure.
The FDA’s AGAMREE approval was the result of data from the VISION DMD Phase 2b trial. When compared to current steroids (corticosteroids), vamorolone was found to exhibit comparable efficacy and a reduction of adverse events relating to growth, health, and behavior. The Muscular Dystrophy Association is listed as the number 1 voluntary health organization in the United States for people living with muscular dystrophy, ALS, and neuromuscular diseases. The Association has been active for the past 70 years in research, advanced care, and patronage for DMD families. Its mission is to empower people to live more independent lives.
