Innovations in Biotechnology and Medical Sciences

Affordable treatment for Duchenne Muscular Dystrophy (DMD)  


From UPSC perspective, the following things are important :

Prelims level: Duchenne Muscular Dystrophy (DMD)

Mains level: NA

Central Idea

  • Researchers in India are collaborating to develop an affordable treatment for Duchenne Muscular Dystrophy (DMD), a rare and incurable genetic disorder.
  • The Indian Institute of Technology (IIT), Jodhpur, is collaborating to develop affordable therapeutics for DMD.

What is DMD?

  • DMD is a progressive muscle degeneration disorder caused by alterations in the dystrophin protein.
  • It is the most common and fatal type of muscular dystrophy, primarily affecting boys.
  • It leads to progressive muscle degeneration, weakness, and eventually wheelchair dependency, assisted ventilation, and premature death.

Symptoms and Impact of DMD

  • Muscle Weakness: Muscle weakness is the primary symptom of DMD, initially affecting proximal muscles and later distal limb muscles. Difficulties in jumping, running, and walking are common.
  • Other Symptoms: Enlargement of calves, a waddling gait, lumbar lordosis (inward curve of the spine), and later heart and respiratory muscle involvement. Pulmonary function impairment and respiratory failure may occur.

Current Challenges

  • Costly treatment: Current therapeutic options for DMD are minimal and expensive, with costs reaching up to Rs 2-3 crore per child per year.
  • Limited Treatment Options: The treatments are predominantly imported, making them financially unattainable for most families.

Efforts to Develop Affordable Therapeutics

[A] Antisense Oligonucleotide (AON)-Based Therapeutics

  • The IIT Jodhpur researchers are working on enhancing the efficacy of AON-based therapeutics.
  • AONs can mask specific exons in a gene sequence, addressing the challenges faced in DMD patients.
  • Personalized medicine is necessary due to the variations in mutations among DMD patients.

[B] Clinical Trials and Molecular Tags

  • The research team has received approval from the Drugs Controller General of India (DCGI) to conduct multi-centric clinical trials on AON-based exon skipping in DMD patients.
  • They are also working on reducing the therapeutic dose of AON through new molecular tags.

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