Lucknow: Sanjay Gandhi Post Graduate Institute of Medical Sciences (SGPGIMS) has begun providing a ‘Made in India’ oral therapy to children with spinal muscular atrophy (SMA), issuing medicines to the first batch of 15 patients free of cost under the National Policy for Rare Diseases (NPRD).SMA is a rare genetic disorder in which a faulty gene results in a shortage of a protein essential for the survival of motor neurons, leading to progressive muscle weakness and muscle wasting. One of the available treatment options is Risdiplam, an RNA-splicing modifier that acts on RNA to increase production of the protein needed for motor-neuron survival.Prof Kaushik Mandal, head of the Medical Genetics department at SGPGIMS, said the Indian-made version dramatically lowers the cost of treatment. “For a 20-kg child, foreign-made versions of this drug cost about Rs 1-2 crore per year. But the expenditure comes down to Rs 3.5 lakh per annum with the Made in India counterpart, which is up to nearly 57 times cheaper,” he said. He added that distribution to another 50 children is underway and noted that SGPGI is among 15 centres across the country rolling out the medicine to eligible children.Prof Mandal said the move would ease the burden on families managing long-term care and treatment expenses. He cited global prevalence estimates of about one in 10,000 live births, adding that roughly one in 50 people carry the faulty SMA gene without symptoms. He also pointed to the high costs of other modalities, stating that virus-based gene therapy can cost around Rs 18 crore, while intrathecal genetic-modifier injections can cost about Rs 6 crore annually.SGPGIMS director Prof Radha Krishnan Dhiman said the rollout represents a significant step toward making treatment for rare genetic disorders in India more affordable and accessible.
