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.
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.