Rainbow Children's Hospital in Secunderabad, India has successfully administered gene therapy to a child diagnosed with Spinal Muscular Atrophy (SMA), marking the eighth such treatment carried out at the facility. The therapy was conducted under the supervision of Dr. Ramesh Konanki, Consultant Pediatric Neurologist at the hospital.
SMA is a devastating genetic neuromuscular condition that affects approximately one in 10,000 children globally. In India alone, an estimated 1,000 children are currently battling this disease, which progressively destroys motor neurons controlling muscle movement.
Breakthrough Gene Therapy Treatment
The patient received Zolgensma, a one-time gene therapy specifically developed to treat SMA by replacing the defective SMN1 gene responsible for the condition. The therapy is administered through an adeno-associated viral vector, enabling the production of the SMN protein essential for the survival of motor neurons and muscle function.
Priced at approximately Rs 14 crore (USD 1.7 million) per dose, Zolgensma is considered one of the most expensive therapies currently available worldwide. Despite the high cost, the treatment offers hope for children with this otherwise progressive and often fatal condition.
Specialized Care Requirements
Rainbow Children's Hospital in Secunderabad is among the few centers in India equipped with the infrastructure and multidisciplinary expertise necessary to deliver such cutting-edge gene therapies. Following administration of Zolgensma, patients require continuous monitoring by a specialized team and weekly laboratory investigations to identify potential adverse events and enable early intervention.
"This treatment has given our family renewed hope," said Vineet Chaudary, father of the child who received the therapy. "We are grateful to the entire team at Rainbow Children's Hospital for their professionalism and support. We also extend our heartfelt thanks to all the well-wishers and donors who came forward to help us make this possible."
SMA Treatment Landscape
SMA is caused by a mutation in the survival motor neuron 1 (SMN1) gene, which leads to insufficient production of the SMN protein. Without adequate levels of this protein, motor neurons die, resulting in progressive muscle weakness and atrophy.
Before the advent of gene therapies like Zolgensma, treatment options for SMA were limited and primarily focused on managing symptoms rather than addressing the underlying genetic cause. The approval of gene therapy represents a paradigm shift in the management of this devastating condition.
Rainbow Children's Hospital's growing experience with administering Zolgensma highlights the increasing accessibility of advanced genetic treatments in India, though significant challenges remain regarding the high cost and specialized care requirements associated with such therapies.
