What is Zolgensma?

Spinal Muscular Atrophy (SMA) is a rare genetic disease affecting muscle function. In India, treatment options are limited, mainly due to high costs. The gene therapy drug, Zolgensma, costs around 16 crore rupees. Despite this, some families are finding ways to access treatment.

About Spinal Muscular Atrophy (SMA)

SMA is a genetic disorder that leads to the loss of motor neurons in the spinal cord. This results in muscle weakness and atrophy. Symptoms often appear in infancy. Children may struggle with basic movements like lifting their heads or crawling. Early diagnosis is crucial for effective treatment.

About Zolgensma

Zolgensma (onasemnogene abeparvovec-xioi) is a revolutionary gene therapy for SMA. It addresses the root cause by replacing the missing or defective SMN1 gene. This therapy can improve motor function in affected children. Although it is costly, it represents hope for many families.

SMA is not as rare as once thought. The global prevalence is estimated at 1 in 8,000 births. Increased awareness is crucial. About SMA can lead to earlier diagnosis and treatment, improving outcomes for affected children.

Challenges in Accessing Treatment

The high cost of Zolgensma poses an important barrier for many families in India. Limited access to healthcare facilities equipped to administer this treatment further complicates the situation. Crowdfunding and community support are vital for families seeking this life-saving therapy.

Advancements in SMA research continue to emerge. Ongoing efforts aim to reduce treatment costs and improve accessibility. Increased awareness and advocacy are essential for enhancing support systems for families affected by SMA.

Month: Current Affairs - November, 2024

Category: Science & Technology Current Affairs