There’s an unobtrusive bungalow in a quiet leafy neighbourhood in North Bangalore. From the outside it looks no different from any of the other houses in the lane. Once inside, you find yourself transported to the set of a science fiction movie. Hidden behind the deceptively domestic exterior is a state-of-the-art genetics laboratory, complete with all sorts of sophisticated equipment — cryo-facilities, autoclaves, air showers and clean rooms — the likes of which would have been the envy of a pharmaceutical R&D facility anywhere in the world.
This is the DART laboratory, where one of the most remarkable stories of Indian medical ingenuity is taking place. This is the story of one family’s heroic effort to find the cure for an incurable disease, racing against time to try and save their son’s life before it was too late.
Movin, Ravdeep and Karan with Rahul at the DART lab.
Duchenne’s Muscular Dystrophy (DMD) is a degenerative genetic disease that afflicts 1 in every 3500 male children. If you have Duchenne’s you are unlikely to live past your twenties. DMD is incurable, but with advances in genetic science and a better understanding of how the disease is caused, scientists are experimenting with radical new treatments to try and slow it down.
But these are radical new therapies largely being developed on an experimental basis in laboratories in the developed world. How can we bring that science to India so that we can save our children from certain death?
Additional reading:
If you want to understand a little more about how exon skipping works for DMD patients this website explains it simply, with pictures.
For more on DART and the wonderful work they are doing for patients suffering from DMD and other similar genetic diseases please visit their website. Every year there is a rare disease symposium that discusses issues facing not just DMD but a whole host of other rare diseases. If you are interested read about the 2016 symposium here and the 2017 symposium, here.
Thanks to Ravdeep Singh Anand, Movin Anand and Karanveer Singh Anand, and Professor Vijay Chandru.