Rare gene therapy performed at children’s hospital in Hyderabad

Our Bureau Hyderabad | Updated on June 12, 2021

Three-year-old patient suffering from Spinal Muscular Atrophy, a progressive neuromuscular disease, was given the therapy

The doctors at Rainbow Children’s Hospital here have performed a rare gene therapy for the third time in the last ten months.

According to a release, three-year-old Ayaansh Gupta, who is suffering from Spinal Muscular Atrophy (SMA), was given Zolgensma injection on June 9 under the supervision of Ramesh Konanki, Consultant Pediatric Neurologist.

Zolgensma is the world’s most expensive drug, currently not available in India. It has been imported from the US at a cost of Rs 16 crore.

Spinal Muscular Atrophy is a progressive neuromuscular disease caused due to a defect in the SMN1 gene.

The affected children develop muscle weakness involving the upper and lower limbs initially, but over time develop difficulty in breathing and swallowing.

SMA affects one in 10,000 children, and there are nearly 800 children suffering with SMA in India.

Published on June 12, 2021

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