Hyderabad-based Rainbow Children’s Hospital, which is the best pediatric hospital in the country, has added one more feather to its cap. The hospital performed a rare gene therapy for the third time in last 14 months.
Master. Ayaansh Gupta, aged 3 years, son of Mr. Yogesh Gupta and Mrs. Rupal Gupta, who is suffering from Spinal Muscular Atrophy (SMA) was given ZOLGENSMA a injection on June 9 2021, at Rainbow Children’s Hospital, Secunderabad, under the supervision of Dr. Ramesh Konanki, Consultant Pediatric Neurologist. ZOLGENSMA is the world’s most expensive drug, currently not available in India. It has been imported from USA at a cost of USD 2,125,000 (INR. 16 Crores).
Rainbow Children’s Hospital is one of the select few hospitals in the country, and the only centre in Telangana and Andhra Pradesh, to have facilities and expertise to administer gene therapy.
Spinal Muscular Atrophy is a progressive neuromuscular disease caused due to defect in SMN1 gene. The affected children develop muscle weakness involving upper and lower limbs initially, but over time develop breathing difficulty and swallowing difficulty. SMA affects 1 in 10,000 children generally, and there are nearly 800 children suffering with SMA in India currently, and thrice as many children die before they even reach their 2nd birthday.
ZOLGENSMA is a single dose intravenous injection gene therapy, in which the defective SMN1 gene is replaced through adenoviral vector. Earlier, two children have been given ZOLGENSMA at Rainbow Children’s Hospital, Secunderabad, in August 2020 and April 2021, and the medicine was provided by Novartis free of cost, on compassionate grounds. Both these children are recovering quite well and making progress in their physical growth and development.
Elaborating details, Dr. Ramesh Konanki stated that currently, there are three proven treatments for children suffering from SMA:
“They have to be given either of ZOLGENSMA, SPINRAZA and RISDIPLAM and unfortunately none of these medicines are currently available in India, and all are hugely expensive. SPINRAZA and RISDIPLAM need to be taken life-long, and costs around Rs 40-70 Lakh per year. 100s of children affected with SMA do not have access to any of these treatments due to the mammoth costs involved for treatment,” Dr Ramesh said.
The Consultant Pediatric Neurologist, on behalf of the medical community and the SMA community (CureSMA group) request Central and State governments to negotiate with respective pharma companies and make these medicines available at affordable price
Further, Dr. Ramesh Konanki desired policy makers to encourage research into treatments for rare diseases through provision of research funding and also provision of medical insurance for frequent hospitalization and surgeries.
Ayaansh’s parents happiness knew no bounds as their son was administered the life saving medicine at last. A visibly elated Ayaansh’s father Yogesh Gupta said, “We are thankful to the team at Rainbow Children’s Hospital, for taking good care of Ayaansh. Grateful to all the donors and ImpactGuru, who have given Ayaansh a gift of life by getting him the world’s most expensive medicine. Please continue praying for Ayaansh’s quick recovery. We would never have reached so far without the support of you all.”
“The parents were able to arrange the amount after going through crowd funding by ImpactGuru.com, and the union finance ministry has waived off the tax worth around 6 crores Rainbow hospitals appreciate the perseverance and courage shown Ayaansh’ parents for the relentless efforts to save their son’s life. Also, I would like to request the Indian government to make this injection available in India for a price that can be afforded by every parent,” said Dr. Preetham Kumar, Consultant – Pediatrician & Pediatric Intensive Care, Rainbow Children’s Hospital Secunderabad.
Bollywood Celebrities and Cricketers have also done their bit
A large number of Bollywood Celebrities namely Ajay Devgan, Anil Kapoor, Shraddha Kapoor, Alia Bhatt, Rajkumar Rao, Karthik Aryan, Sara Ali Khan, Arjun Kapoor, Anurag Basu and Cricketers R Ashwin, Washington Sundar, Dinesh Karthik amongst others shared Ayaansh’s ImpactGuru fundraiser on their social media pages to reach out to their millions of followers. This resulted in a massive spurt of donations. Major celebrity donors include Virat Kohli, Anushka Sharma, Arjun Kapoor, Sara Ali Khan etc. Major corporate donors include Dharma Productions, T-Series, Cipla among many others.