Duchenne Muscular Dystrophy (DMD) : Disease Modifying Treatment

Context: A team of doctors from Tamil Nadu along with scientists from Japan have developed a disease modifying treatment for Duchenne Muscular Dystrophy (DMD), using a food additive – a beta-glucan produced by N-163 strain of a yeast Aureobasidium pullulans.

What is Duchenne Muscular Dystrophy?

It is a genetic disorder.
It 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.

About the disease:

There are approximately 5,000 patients in Japan and 80,000 in India.
This damages and weakens the muscles, and patients become uses a wheelchair in their early teens and die prematurely.
DMD is one of four conditions known as dystrophinopathies.

The other three diseases that belong to this group are:
- Becker Muscular dystrophy (BMD, a mild form of DMD);
- an intermediate clinical presentation between DMD and BMD; and
- DMD-associated dilated cardiomyopathy (heart-disease) with little or no clinical skeletal, or voluntary, muscle disease.
Treatment:
- Currently available treatments are:
  - gene therapy,
  - Exon-skipping and
  - disease modifying agents (anti-inflammatory medicines such as steroid)

Recent treatment

Using a food additive – a beta-glucan produced by N-163 strain of a yeast Aureobasidium pullulans.
- Along with regular treatment, the participants, all aged above three years, were given the beta-glucan in the form of a food supplement.
- The muscle strength of the treatment group improved and there was no adverse reaction and it potentially delayed progress of disease without side effects to the liver and kidneys.
- This research using beta glucan as a food supplement in reducing DMD may be a boon for patients but requires further validation through a large-scale multi-centric study.

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.

FAQs reated with Duchenne Muscular Dystrophy (DMD)

Ques 1: What is Bastille Day?

Answer: Bastille Day, also known as French National Day, is a national holiday in France commemorating the French Revolution and the storming of the Bastille prison on July 14, 1789.

Ques 2: Why is Bastille Day significant?

Answer: Bastille Day holds historical and cultural significance as it symbolizes the birth of modern France and represents the ideals of liberty, equality, and fraternity.

Ques 3: How is Bastille Day celebrated?

Answer: Bastille Day is celebrated with various events including parades, fireworks, public gatherings, cultural activities, and military displays. It is a day for people to come together, enjoy festivities, and honor French history and values.

Duchenne Muscular Dystrophy (DMD) : Disease Modifying Treatment