According to a story from PR Newswire, the specialty drug development company Mallinckrodt recently announced that enrollment for trials of its experimental medication MNK-1411 has started. The drug will be tested as a treatment for Duchenne muscular dystrophy in patients aged 4 to 8 years. MNK-1411 has already received Orphan Medicinal Products designation from the European Medicines Agency for the treatment of this disease.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is one of the most severe types of muscular dystrophy; it is also the most common. The disease is characterized by progressively worsening muscle weakness in boys that usually begins to appear around age four. Duchenne muscular dystrophy is caused by a mutation that prevents the body from producing dystrophin, a compound that is essential for the maintenance of muscular structure. Symptoms include muscle atrophy, which affects the pelvic area, hips, calves, and shoulders first. Other muscles, including respiratory muscles are affected as the disease progresses. Other symptoms include fatigue, falling, poor motor skills, scoliosis, loss of walking ability, mental health issues, and muscular deformities. Treatment may include physical therapy and medication, which can help slow the progression of the disease somewhat. Life expectancy averages around 25 years, but can be longer with top-of-the-line care. To learn more about Duchenne muscular dystrophy, click here.
About The Clinical Trial
The trial is unique in that MNK-1411 will be tested on patients in a very specific age range (4-8 years). The researchers aim to recruit a total of 130 patients for the study. The patients will receive dosages of MNK-1411 based on weight, to be administered twice a week for a period of 24 weeks. At the end of the treatment period, response will be based on data from several timed function tests.
Duchenne muscular dystrophy is currently an unmet medical need; at this juncture, there is currently no available cure for the disease, nor is there a treatment that can stop progression of the disease. While it will be some time before data from this trial will become available, hopefully MNK-1411 will be able to prove itself as a viable treatment.