Jaipur (Rajasthan): Six-month-old girl, Noor Fatima from Rajasthan's Bikaner district, has died, as her parents couldn't afford to get the Rs 16 crore injection - Zolgensma, a gene therapy medication that is used to treat spinal muscular atrophy (SMA).
The killer muscular disorder progressively destroys nerve cells in the brain stem and spinal cord that control speaking, walking, breathing, and swallowing. Due to lack of money and treatment, the girl died Tuesday morning. However, some social organizations had come forward to make donations to Noor Fatima. Recently, Noor Fatima's uncle Inayat Ali, in his interaction with ETV Bharat, had appealed to Prime Minister Narendra Modi to save the girl's life.
'Zolgensma' is the world's most expensive drug and is currently not available in India. It is being imported from the US at a cost of Rs 16 crores. 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 breathing difficulty and swallowing difficulty.
SMA affects one in 10,000 children generally, and there are nearly 800 children suffering from SMA in India currently, and thrice as many children die before they even reach their second birthday. It is a single-dose intravenous injection gene therapy in which the defective SMN1 gene is replaced through an adenoviral vector.
Read: Pune couple resorts to crowdfunding for treatment of son suffering from SMA
