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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...

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 

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