Affan, seven, and Erhan, five, have Spinal Muscular Atrophy - a rare genetic disorder that causes muscle deterioration and affects breathing. Physiotherapy appointments alone cost the family 40,000 rupees a month, and their sons need constant care as they cannot sit, stand or walk independently.
Like them, many parents in India cannot afford to buy Zolgensma and other SMA drugs. While there is no official data on the number of Indians with the disease, existing literature shows that SMA affects- according to one study, one in 38 Indians are carriers of the faulty gene that causes SMA, compared with 1 in 50 people in the West.
India has a rare diseases policy which aims to provide financial help, but experts say it hasn't been implemented properly yet. are less severe, but in these cases too gradual muscle deterioration is imminent, shortening the person's lifespan. And factors like age or health issues might render a person ineligible for these programmes. Novartis, which manufactures Zolgensma, offers the drug to children under two years through a lottery system.
- an expert panel it set up to implement India's rare diseases policy - to speak to manufacturers to see if SMA medicines could be procured at lower prices.it would cover the cost of Zolgensma for infants with the most severe cases of SMA after dozens of families fought and won court cases. The Brazilian government has struck a confidential deal with Novartis to buy the drug at a reduced price and pay for it in instalments, the New York Times reported.