LUCKNOW, Aug. 28 -- In a major boost to the treatment of rare genetic disorders, Sanjay Gandhi Post-Graduate Institute of Medical Sciences (SGPGIMS), Lucknow, on Thursday launched the first "Make in India" oral RNA splicing modifier therapy for children suffering from spinal muscular atrophy (SMA) under the National Policy for Rare Diseases (NPRD). Conventional SMA therapies are prohibitively expensive. Gene therapy can cost around Rs.18 crore, while intrathecal genetic-modifier treatment costs nearly Rs.6 crore annually. The India-made formulation brings the annual cost down to about Rs.3.5 lakh-nearly 50 times lower. The institute distributed the first batch of Risdiplam (medication used to treat SMA) free of cost to 15 children with SMA at the department of medical genetics. The SGPGIMS has more than 100 registered SMA patients with over 50 children awaiting treatment. PGI director Dr RK Dhiman congratulated the medical genetics team and distributed the medicines. He said the initiative marks a significant milestone in rare-disease care. SMA, which affects around one in 10,000 live births globally, is a genetic disorder in which motor neurons in the spinal cord progressively degenerate, causing muscle weakness. Affected children develop complications including scoliosis, respiratory difficulties and reduced speech volume. However, their intelligence and cognitive abilities remain intact. The NPRD, implemented by the Union health ministry, seeks to make treatment for rare diseases accessible amid mounting pressure over the high cost of imported therapies. htc...