TATA EDUCATION AND DEVELOPMENT TRUST

Tripura SMA Type 1 Case Highlights India's Rare Disease Funding Gap as Risdiplam Vials Supplied, Zolgensma Awaits DCGI Approval

- A 22-month-old Tripura child with Spinal Muscular Atrophy Type 1 has received 12 vials of Risdiplam from a designated Centre of Excellence in Dibrugarh. - The supply, valued at about Rs 2.29 lakh, is expected to cover roughly eight months of treatment under India's Rare Disease Programme. - AIIMS New Delhi experts recommended Zolgensma gene therapy, but the assessment noted the drug was still awaiting Drug Controller General of India approval. - The case exposes the gap between the Rs 50 lakh NPRD assistance cap and gene therapies priced at Rs 16-18 crore, with no official crowdfunding listing yet verified.