Indians who need a $2.1m drug to fight rare disease
Their condition is Spinal Muscular Atrophy (SMA), a rare genetic disorder that causes muscle deterioration and affects breathing. Their sons cannot sit, stand or walk independently, so they need constant care due to their physical disabilities. Physiotherapy alone costs the family 40,000 rupees ($480; £395) a month.
Zeba Gufran, their mother, says that they want to try gene therapy for their sons, but a single dose costs around 175 million rupees ($2.1 million; £1.7 million).
The Gufrans want to try Zolgensma gene therapy, which is one of the most expensive drugs on the market. A one-time dose is given to children under two – but the Gufrans hope for a miracle.
In India, many parents cannot afford Zolgensma and other SMA drugs. The number of Indians with the disease is unknown, but existing literature indicates that nearly 1 in 10,000 live-born babies suffer from SMA – according to one study, 1 in 38 Indians carry the faulty gene that causes the disease, compared to 1 in 50 Westerners.
Some countries cover the cost of treating rare diseases, even though they are expensive everywhere. SMA medicines are available through the National Health Service in the UK, while Australia offers subsidised access to expensive lifesaving drugs to eligible patients.
Crowdfunding is often used by Indian patients to access these treatments. Patients and their families are putting pressure on the federal government to lower the cost of these drugs as the spotlight grows on rare genetic diseases.