Treatment for Duchenne Muscular Dystrophy | 18 Jul 2023

Source: TH

Why in News?

A collaborative effort between doctors from Tamil Nadu, India, and scientists from Japan has resulted in the development of a disease-modifying treatment for Duchenne Muscular Dystrophy (DMD).

What is the Disease Modifying Treatment for DMD and Major Findings?

  • This treatment utilizes a food additive called beta-glucan, derived from the N-163 strain of the yeast Aureobasidium pullulans.
  • The six-month-long clinical study involved 27 children with DMD, of which 18 were in the treatment group and 9 in the control group.
    • In addition to regular treatment, all participants (age >3) received the beta-glucan as a food supplement.
  • The study yielded the following notable findings:
    • Reduction in Muscle Weakness and Damage: Evidence indicated a reduction in muscle weakness and damage among the treatment group.
      • It also led to improved Muscle Strength.
    • Safety and Lack of Adverse Reactions: No adverse reactions were observed in the participants, and the treatment showed no side effects on the liver and kidneys.

Note: Beta-glucan is a polysaccharide (a complex sugar) that has anti-inflammatory, immunomodulatory and antioxidant properties.

What is Duchenne Muscular Dystrophy?

  • About:
    • Duchenne Muscular Dystrophy (DMD) is a rare genetic disease characterized by the inability of muscles to produce dystrophin, an enzyme that aids in muscle wear and tear as well as its regeneration.
      • It affects only male children.
    • The absence of dystrophin leads to muscle damage, resulting in muscle weakness and ultimately wheelchair-bound conditions in early teens and consequent premature deaths.
  • Common Symptoms:
    • Progressive muscle weakness and atrophy (loss of muscle bulk) that begins in the legs and pelvis and later affects the arms, neck and other areas of the body.
    • Difficulty walking, running, jumping, climbing stairs and getting up from a lying or sitting position.
    • Frequent falls, waddling gait (abnormal walking pattern) and toe walking.
  • Prevalence:
    • According to a 2020 study on the global epidemiology of DMD, the pooled global DMD prevalence was 7.1 cases per 100,000 males and 2.8 cases per 100,000 in the general population.
    • Also, it has approximately 5,000 patients in Japan and 80,000 in India.
  • Current Treatments:
    • Currently, there is no known cure for DMD. Treatment aims to control symptoms to improve quality of life.
    • Available treatments for DMD include gene therapy, exon-skipping, and disease-modifying agents such as anti-inflammatory medications and steroids.