Muscular Dystrophy (MD) is a genetic disease caused by progressive degeneration and weakness of the muscles, which arises due to mutations or deletions in the MD gene on the X chromosome, affecting any of its 79 exons. MD results in lots of problems like difficulty in sitting, standing, and walking. If children succumb to this problem, they can hardly live for 20 years. However, if they are lucky enough or have access to very good medical facilities, they may live up to 40 years, but now more than that. As per the WHO report, 1 in every 3500 people is affected with MD, with 54 babies being born every day with this disorder! With such high number of Indians (almost 8-10 lakhs) suffering from MD, it definitely calls for a good amount of attention!
Muscular Dystrophy – No treatment in India yet!
With such huge numbers of Indians suffering from the disease, there are only a handful who have the capacity to travel abroad and get the right treatment. Living in India, patients have a very small hope of getting treated because of the lack of drugs available; and bringing in drugs from countries abroad can be an extremely costly affair. There is no cure or treatment in India at present for this disease; in spite of the large number of people suffering from it. However, the disorder has now come into the limelight, which is why scientists in three different institutes in Kolkata and Bangalore (IACS, ICH, and DART) have started working on the development of a medicine to treat this disease. Out of the three, DART has already conducted a successful trial of an RNA-based molecule on a patient, with permission from the ICMR; and has also applied for further clinical trials of the same to the DCGI. But, this seems no good for those currently suffering from the disease. This is because, even if approved, the drug will only be available in the market in the next 10-12 years. After all, drug development is a very lengthy process, involving lots of trials and approvals.
Muscular Dystrophy – The expenses and extensiveness involved
Looking at this, the parents of almost every child suffering from MD want their child to be a part of these clinical trials with the hope of being treated effectively for the disease. These trials are the only hope for such parents and children. This is because there are many drugs available abroad; for example, the USA drug Exondy51; but such drugs cost Rs. 2-3 crores annually, with one injection being given per week, for 52 weeks! This amount is generally not in the range of an average Indian family. Moreover, the overall management of this disease is extensive, including checks on a regular basis from a wide range of specialists and constant investment towards travelling, accommodation, and medicine in upcoming novel therapies. Then, there are wheelchairs, hists, calipers, transfer boards, leg braces, knee braces, and all sorts of devices used by such patients to keep them at ease.
No one drug suits all!
Apart from all of the above, one other consideration to make is that there are nine different types of MD, with each targeting a different exon in the X chromosome. Thus, there can be no “one solution” for all types of MD. Every different type of MD requires a different kind of medicine; so you can imagine the huge costs involved in the drug development of each drug!
However, we cannot let lakhs of our countrymates succumb to death only because of one particular disorder. We strive to work towards achieving Made-In-India drugs to help such patients, no matter how much time or money it takes. Join hands and put in your efforts and knowledge too by achieving the best clinical research training in Bangalore from Avigna Clinical Research Institute, so that you are eligible to work with professional scientists in our country, and bring about a variety of positive changes in the health sector of India.