Meerut (Uttar Pradesh): Ishani, a 21-month-old toddler from Meerut in Uttar Pradesh, who is battling a rare disease called Spinal Muscular Atrophy (SMA), has been administered the life-saving drug 'Zolgensma' at AIIMS Delhi on Saturday. The girl's parents, who were unable to afford the world's most expensive drug, had resorted to crowdfunding in a bid to save their daughter. However, the drugmaker 'Novartis', which allocates around 100 doses of the medicine free of cost through its global Managed Access Program (MAP), provided the medicine to the toddler, with the Indian Government exempting the tax of Rs 6 crore. The actual cost of the drug is Rs 16 crores, but including taxes, it swells to Rs 22 crore in India. The injection has been administered to the baby girl and she will be under doctors' supervision for three months.
Earlier, after All India Institute Of Medical Science (AIIMS) Delhi had confirmed that the girl had been ailing from SMA type 2 fatal disease, the doctors had advised her parents to buy Zolgensma. As it was impossible for Ishani's parents to arrange such a hefty amount, they took to social media and also appealed to Prime Minister Narendra Modi for assistance. The gene responsible for the disease disrupts the production of proteins which is necessary for the smooth functioning of the nervous system in the body, resulting in nervous system damage. The disease kills the affected infants, usually before they turn two years old.
Read: Infant who needed Rs 16 crore injection dies
Zolgensma, a single-dose intravenous injection gene therapy in which the defective SMN1 gene is replaced through an adenoviral vector, is currently not available in India and is imported from the US. Spinal Muscular Atrophy is a progressive neuromuscular disease caused due to a defect in the SMN1 gene. The affected children develop muscle weakness involving upper and lower limbs initially, but over time develop difficulties in breathing and swallowing. SMA affects one in 10,000 children on an average. There are nearly 800 children suffering from SMA in India.
