Indo-Japanese researchers develop a treatment for Duchenne’s Muscular Dystrophy

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

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About Duchenne Muscular Dystrophy (DMD)

  • DMD is a rare genetic disease that affects only male children.
  • There are approximately 5,000 patients in Japan and 80,000 in India.
  • Muscles need lubricant. Dystrophin, an enzyme secreted in the muscles, helps in wear and tear and regeneration of muscles.
  • Because of the genetic disorder, muscles cannot produce dystrophin.
  • This damages and weakens the muscles, and patients become wheelchair-bound in their early teens and die prematurely.
  • Boys born before 1970 had a median lifespan of around 18 years.
  • After anti-inflammatory medications and steroids, longevity increased.
  • Disease-modifying treatments have prolonged the lifespan but despite that, patients die when they are aged 28 to 30 years.
  • Currently, available treatments are gene therapy, Exon-skipping and disease-modifying agents (anti-inflammatory medicines such as steroids).
  • There was no adverse reaction in the participants and it potentially delayed the progress of the disease without side effects to the liver and kidneys.

Prelims Practice MCQ

Consider the following statements regarding the Duchenne Muscular Dystrophy (DMD)

1.      It is a genetic rare disease

2.      It affects only male children

3.      It is caused by reduced or no production of dystrophin enzyme

4.      There is no treatment available for this disease

How many of the above statement/s is/are correct

(a)    Only one

(b)   Only two

(c)    Only three

(d)   All four

Scroll down for answer










Ans. C

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