In a recent collaboration between medical practitioners from Tamil Nadu, India and researchers from Japan, a new disease-modifying treatment for Duchenne Muscular Dystrophy (DMD) has been innovatively developed. This innovative treatment makes use of a food additive named beta-glucan, derived from the yeast strain N-163 of Aureobasidium pullulans.
Description of the Disease Modifying Treatment for DMD
The clinical study, which spanned over six months, involved 27 children diagnosed with DMD. The participants were divided into two groups – 18 patients in the treatment group while 9 received regular treatment serving as the control group. To supplement their regular treatment, all children aged over three years were given beta-glucan.
Main outcomes of the Beta-glucan treatment
Two remarkable outcomes became evident from the study. Firstly, the experimental treatment group showcased a decline in muscle weakness and damage, leading to an improvement in their muscular strength. Secondly, the participants did not experience any adverse reactions or side effects on their liver and kidneys, proving the safety of the treatment. It’s important to note that beta-glucan is a complex sugar, known for its anti-inflammatory, immunomodulatory, and antioxidant properties.
Duchenne Muscular Dystrophy – an Overview
Duchenne Muscular Dystrophy or DMD is a rare genetic disorder characterized by the failure of muscles to produce dystrophin, a critical enzyme for muscle repair and regeneration. This condition affects male children exclusively, leading to muscle damage, weakness, and eventually wheelchair dependency in early adolescence, followed by premature death.
Common symptoms of DMD
The disease presents with progressive muscle weakness and atrophy initially appearing in the legs and pelvis, subsequently spreading to the arms, neck, and other parts of the body. Patients often find it challenging to perform basic tasks such as walking, running, jumping, climbing stairs, or even transitioning from lying or sitting postures. Further signs include frequent falls, toe walking, and an abnormal walking pattern known as a waddling gait.
Global Prevalence of DMD
Based on a 2020 study on DMD’s global epidemiology, the disease’s worldwide prevalence was found to be 7.1 cases per 100,000 males and 2.8 cases per 100,000 in the general population. Notably, Japan records around 5,000 DMD patients, while India has approximately 80,000 cases.
Contemporary Treatment Techniques
Despite the lack of a definitive cure for DMD, current treatments aim to manage the symptoms thereby enhancing the patient’s quality of life. Current therapeutic techniques for DMD include gene therapy, exon-skipping techniques, and disease-modifying agents such as anti-inflammatory drugs and steroids.
