Boy gets Rs 17cr life-saving gene therapy for rare SMA type 1

A 16-month-old boy, Deen Mohammad from East Midnapore, diagnosed with spinal muscular atrophy (SMA), recently received lifesaving gene therapy with Zolgensma at Peerless Hospital.

- Ashok Chatterjee

SMA, a rare genetic disorder affecting motor neurons, causes progressive muscle weakness and is typically diagnosed in infancy. The Rs 17.5-crore therapy, which Deen received for free under Novartis's Managed Access Programme, is the world's most expensive single-dose treatment.

The father abandoned the mother and the baby after he was diagnosed with SMA. The baby lives in his maternal home with his mother and grandmother.

Diagnosed by Dr Sanjukta Dey at six months, Deen's case required extensive paperwork, supported by Cure SMA Foundation of India, to secure the treatment. Moumita Ghosh, cofounder of the foundation, expressed gratitude towards Novartis and Peerless Hospital for providing the drug and called on the government to improve accessibility for other families. Dr Dey highlighted the need for early detection initiatives and more funding under the Rare Disease Policy, as SMA may become the second most common genetic disorder in India.

However, it's now not considered so rare as the worldwide prevalence is said to be 1 in 8,000.