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States » SouthPosted at: May 22 2025 1:06AM


Hyd: Rainbow Children’s Hospital completes eighth gene therapy for Spinal Muscular Atrophy

Hyderabad, May 21 (UNI) Rainbow Children’s Hospital (RCH( on Wednesday said it has successfully administered ‘gene therapy’ to a child diagnosed with Spinal Muscular Atrophy (SMA), marking the eighth such treatment carried out at the hospital, a significant milestone in paediatric healthcare.
The therapy was administered under the supervision of Dr. Ramesh Konanki, Consultant Pediatric Neurologist, Rainbow Children’s Hospital, Secunderabad, the hospital said in a release here.
As part of the treatment protocol, the child received Zolgensma, a one-time gene therapy specifically developed to treat Spinal Muscular Atrophy (SMA) by replacing the defective SMN1 gene responsible for the condition.
Administered through an adeno-associated viral vector, the therapy enables the production of the SMN protein, which is essential for survival of motor neurons and thus the muscle function.
With a cost of around Rs 14 crore per dose, Zolgensma is considered one of the most expensive therapies currently available worldwide.
SMA is a devastating genetic neuromuscular condition that affects about 1 in 10,000 children globally.
In India alone, it is estimated that nearly 1000 children are currently battling this disease. Without timely intervention, many affected infants do not live beyond their second birthday, as the disease is relentlessly progressive. UNI KNR SSP
Tags: #Hyd: Rainbow Children’s Hospital completes eighth gene therapy for Spinal Muscular Atrophy
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