Daily Current Affairs : 14-July-2023

Duchenne Muscular Dystrophy (DMD) is a genetic disorder that leads to progressive muscle degeneration and weakness due to a deficiency in the protein dystrophin, which plays a crucial role in maintaining muscle cell integrity. Recently, researchers have made significant progress in developing a disease-modifying treatment for DMD using a food supplement known as beta-glucan. It will explore DMD, its symptoms, existing treatments, and the promising potential of beta-glucan as a therapeutic option.

Understanding Duchenne Muscular Dystrophy (DMD)

DMD is a genetic disorder that affects the production of the dystrophin protein, leading to muscle degeneration and weakness over time. The disease primarily affects boys, though girls can also be affected in rare cases. Symptoms of DMD typically appear in early childhood, usually between the ages of 2 and 3. The condition is characterized by the following:

  1. Muscle Weakness: The principal symptom of DMD is muscle weakness, which initially affects the proximal muscles near the core of the body and later progresses to the distal limb muscles near the extremities.
  2. Mobility Difficulties: Children with DMD may have difficulty jumping, running, and walking. They may exhibit a waddling gait, an inward curve of the spine (lumbar lordosis), and experience calf enlargement.
  3. Cardiac and Respiratory Complications: As the disease progresses, it also affects the heart and respiratory muscles, leading to impaired pulmonary function and potentially acute respiratory failure.
  4. Genetic Basis: DMD is caused by a flawed gene on the X chromosome. Carrier females have one normal and one abnormal dystrophin gene but may not show symptoms themselves.
Existing Treatments for DMD

Over the years, advancements in medical research have led to various treatments for DMD aimed at improving quality of life and slowing disease progression. The following treatment options are currently available:

  1. Gene Therapy: Gene therapy seeks to introduce functional copies of the dystrophin gene into the affected cells, aiming to restore dystrophin production. It holds promise, but more research is needed to make it widely available.
  2. Exon-Skipping: Exon-skipping therapies attempt to correct genetic mutations by “skipping” specific sections (exons) of the dystrophin gene during protein synthesis. This approach helps produce a partially functional dystrophin protein.
  3. Disease-Modifying Agents: These include anti-inflammatory medicines, such as steroids, which can help manage symptoms and slow down disease progression.
Recent Advancement: Beta-Glucan as a Potential Treatment

In a recent study, researchers explored the use of a food supplement called beta-glucan produced by the N-163 strain of the yeast Aureobasidium pullulans as a potential treatment for DMD. Participants above the age of three years were given beta-glucan in addition to their regular treatments. The results showed:

  • Improved Muscle Strength: The participants who received beta-glucan demonstrated improved muscle strength, which is a promising sign of its potential effectiveness in managing DMD.
  • No Adverse Reactions: The beta-glucan treatment did not show any adverse reactions, making it a safer alternative for managing the disease.
  • Delayed Progression of Disease: The researchers observed that beta-glucan had the potential to delay disease progression without causing side effects to the liver and kidneys.

Important Points:

  • Duchenne Muscular Dystrophy (DMD) is a genetic disorder characterized by progressive muscle degeneration and weakness due to a deficiency in the protein dystrophin.
  • Symptoms of DMD typically appear in early childhood and primarily affect boys, although girls can be affected in rare cases.
  • Muscle weakness is the main symptom, starting with proximal muscles near the core of the body and later affecting distal limb muscles near the extremities.
  • Mobility difficulties, such as difficulty jumping, running, and walking, along with calf enlargement and lumbar lordosis, are common symptoms.
  • DMD can also lead to cardiac and respiratory complications, potentially causing acute respiratory failure.
  • The disease is caused by a flawed gene on the X chromosome, and females who carry one normal and one abnormal dystrophin gene may not show symptoms themselves.
  • Current treatments for DMD include gene therapy, exon-skipping, and disease-modifying agents like steroids.
  • A recent advancement in DMD treatment involves using a food supplement called beta-glucan, produced by the yeast Aureobasidium pullulans.
  • Beta-glucan has shown promise as a disease-modifying agent, potentially delaying disease progression and improving muscle strength in DMD patients.
  • The beta-glucan treatment did not result in adverse reactions and could be a safer alternative for managing DMD.
  • Further validation through large-scale multi-centric studies is required to fully understand the effectiveness and safety of beta-glucan as a treatment for DMD.
Why In News

In a groundbreaking development, the newly devised disease modifying treatment for Duchenne Muscular Dystrophy (DMD) has demonstrated remarkable efficacy in halting the progression of the disease and improving the quality of life for affected individuals. With this significant medical advancement, there is newfound hope for those living with DMD and their families, as it opens doors to a brighter and more promising future.

MCQs about Advancements in DMD Treatment

  1. What is the primary symptom of Duchenne Muscular Dystrophy (DMD)?
    A. Calf enlargement
    B. Respiratory difficulties
    C. Muscle weakness
    D. Difficulty jumping
    Correct Answer: C. Muscle weakness
    Explanation: DMD is characterized by progressive muscle degeneration and weakness, which starts with the proximal muscles and later affects the distal limb muscles.
  2. Which treatment approach aims to introduce functional copies of the dystrophin gene into affected cells?
    A. Exon-skipping
    B. Disease-modifying agents
    C. Gene therapy
    D. Beta-glucan
    Correct Answer: C. Gene therapy
    Explanation: Gene therapy seeks to introduce functional copies of the dystrophin gene to restore dystrophin production and potentially slow down disease progression.
  3. what was the role of beta-glucan in managing DMD?
    A. It improved cardiac and respiratory functions.
    B. It delayed disease progression without side effects.
    C. It completely cured DMD in the participants.
    D. It helped in muscle tissue regeneration.
    Correct Answer: B. It delayed disease progression without side effects.
    Explanation: The recent study using beta-glucan as a food supplement showed promising results in potentially delaying the progress of DMD without causing adverse reactions to the liver and kidneys.

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