Indian researchers developing treatment for rare genetic disorder ‘Duchenne Muscular Dystrophy’

Source: The post is based on the article “Indian researchers developing treatment for rare genetic disorder ‘Duchenne Muscular Dystrophy” published in The Hindu on 10th January 2023

What is the News?

Researchers in India are working on developing an affordable treatment for a rare and incurable genetic disorder called Duchenne Muscular Dystrophy with over 5 lakh cases in the country.

What is Duchenne Muscular Dystrophy?

Duchenne muscular dystrophy is a genetic disorder. The disorder is marked by progressive muscle degeneration and weakness due to alterations of a protein called “dystrophin” that helps keep muscle cells intact. 

The condition is predominantly seen in boys, but in rare cases, it can also affect girls.

Symptoms: Muscle weakness is the principal symptom of DMD. It can begin as early as age 2 or 3, first affecting the proximal muscles (those close to the core of the body) and later affecting the distal limb muscles (those close to the extremities). The affected child might have difficulty jumping, running, and walking.

Treatment: Currently, there is no cure for DMD, but improvements in integrative treatment can slow down the disease progression and thereby, extend the life expectancy of DMD patients.

What are the researchers from India developing?

The current therapeutic options available to treat DMD are minimal and highly expensive treatment with costs shooting up to Rs 2-3 crore per child a year and are mostly imported from abroad, accelerating dosing costs and putting them out of reach for most families.

That is why researchers from India are working on an affordable therapeutics for DMD.