Aakancha Shaw, St. Xavier’s College (Kolkata)
Muscular dystrophy implies a group of inherited disorders that can cause loss of muscle tissue, muscle weakness and delayed development. which gets worse over time. Duchenne muscular dystrophy is such muscular dystrophy that worsens quickly unlike the other types of muscular dystrophy. DMD refers to the genetic disorder wherein the body is unable to create dystrophin. Dystrophin is a crucial protein that protects the cardiac and skeletal muscles from injury. This muscular dystrophy disorder is caused by the mutation in the X chromosome and affects only boys. . 1 out of every 3600 male infants suffers from this inherited disorder. Unfortunately, there is no known cure for Duchenne muscular dystrophy (DMD). However, certain glucocorticoid drugs aim to ease symptoms but they have significant side effects.
Post the 24-month clinical trial at Duke Health and other institutions Edward C. Smith, a neurologist, co-director of the Duke Children’s Neuromuscular Program and a clinical investigator in the trial reported that a new drug, Vamorolone is new hope for young boys suffering from Duchenne muscular dystrophy (DMD). Vamorolone, the experimental treatment for DMD was developed by ReveraGen Biopharma. It was found that the drug Vamorolone (anti-inflammatory steroid) could act as an alternative to the glucocorticoids that have abysmal side effects (bone fragility, stunted growth and insulin resistance). These side effects were highly undesirable.
The interim results showed that the drug can significantly reduce the side effects occurring due to the high dosage of steroids like prednisone or deflazacort alongside retaining the therapeutic benefits of the drug. The impact on the bone health of patients taking vamorolone into account showed much less deteriorating and negative impacts. The conventional steroid therapy was known to extend patients’ mobility but the side effects like the inhibition of bone length and development of growth plates in young patients, cannot be ignored. Following the 6-month study, the 46 people who were kept under trial were asked to either opt to prednisone or continue their treatment with vamorolone. All of them opted for Vamorolone following which the study was extended for 2 years. The Phase 2 study is designed to evaluate the efficacy and safety of the drug. The use of Vamorolone showed improvement in the strength and function of the bones with no significant array of side effects. In a presentation at the WMS, Eric Hoffman, the chief executive officer of ReveraGen presented these findings in a detailed manner. Thus, this interim paved a new pathway for the patients affected by this rare disorder.
Chronic Fatigue Syndrome: Post-exertional Malaise studies
BISWADEEP SEN, AMITY UNIVERSITY KOLKATA Persistent fatigue, increasing exercise intolerance, cognitive difficulties, or joint pain are the main symptoms of Post-exertional malaise. It is the pivotal reason for ME /Chronic Fatigue Syndrome. 43 patients were subjected to a Cardiopulmonary exercise test and the data was categorized systematically for analysis. In CPET, the patients have to […]