Lack of treatment, inadequate guidelines and funding are proving to be a major challenge to handle the Duchenne Muscular Dystrophy (DMD) cases in India and globally. So far, no drug has been developed for treatment of this genetic disorder which was detected over two decades ago.
Currently, the only available options to provide some relief to these patients are parent counselling and recommendation of standard of care from the United Parents Projects Muscular Dystrophy (UPPMD). The global body of parents, UPPMD, is associating with clinicians to support the patients suffering from this genetic disorder.
The inadequate knowledge in DMD and lack of a specialization are also proving to be a challenge for physicians and parents, expressed Dr Elizabeth Vroom, chairman, UPPMD at the first Asian Conference on DMD organized here by UPPMD, Healthcare Global Foundation and National Institute of Mental Health and Neuro Sciences (NIMHANS) recently. In spite of the challenges, UPPMD has been able to raise funds to the tune of $12 billion for research to find effective drugs for this disorder, she added.
DMD is the most common childhood disease affecting young boys. The disease affects a child when he barely able to walk. He will require a wheelchair by 12 and succumbs to the disease by 20 years as the respiratory muscles fail to function. One in every 3,500 boys which is an estimated 20,000 babies are annually afflicted by this genetically inherited disorder.
There is presently no cure for DMD. Only steroids like Deflazacort and prednisone which are anti-inflammatory cum immunosuppressants appear to be effective and proving to be a gold standard for the treatment. Clinical trials are with a certain class of drugs help to slow down muscle weakness. According to medical experts, children treated with steroids walk for longer and have improved respiratory function. Studies are on with stem cell research and gene therapy to address the treatment options to stop the progression of some types of muscular dystrophy.
Present treatment is designed to help prevent or reduce deformities in the joints and the spine and to allow people with the disorder to remain mobile as long as possible. These include various types of physical therapy, medications, assistive devices and surgery, stated Dr B S Ajai Kumar, chairman Health Care Global Foundation.
Treatment is aimed at control of symptoms to maximize the quality of life. Physical therapy is proving to be helpful to maintain muscle strength and function. Orthopaedic appliances like braces and wheelchairs are recommended to improve mobility and the ability for self-care, stated Dr Kumar.
The two day conference provided an ideal platform for parents, clinicians and researchers to interact. The international conference had several international speakers delving in the therapeutic approaches, care and management of DMD.