Tripura SMA Type 1 Case Highlights India's Rare Disease Funding Gap as Risdiplam Vials Supplied, Zolgensma Awaits DCGI Approval
核心洞察
A 22-month-old Tripura child with Spinal Muscular Atrophy Type 1 (搜索) 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 (搜索) experts recommended Zolgensma gene therapy, but the assessment noted the drug was still awaiting Drug Controller General of India (搜索) approval.
A 22-month-old child from Hapania, Agartala, diagnosed with Spinal Muscular Atrophy (SMA) Type 1 has become the focal point of a widening debate over how India funds advanced therapies for rare diseases, after she received 12 vials of Risdiplam from a designated Centre of Excellence (CoE) while a gene therapy recommended by AIIMS New Delhi (搜索) remained unapproved in the country.
According to the Tripura Health Department, Manashree Chowdhury's parents approached Chief Minister Dr. Manik Saha during the Mukhyamantri Samipeshu programme in June 2026 seeking government assistance for their daughter's treatment. SMA Type 1 is a rare genetic disorder that affects motor neurons and can severely impair muscle and physical development.
Risdiplam Supplied Under the Rare Disease Programme
Following the family's appeal, Chief Minister Manik Saha wrote a DO letter to Union Health Minister J. P. Nadda on July 9, 2026, seeking possible assistance for the child's treatment. The Union Health Ministry responded on July 24, informing the Chief Minister that the National Policy for Rare Diseases (NPRD), 2021 provides financial assistance of up to Rs 50 lakh per patient for treatment of identified rare diseases at designated Centres of Excellence.
The Ministry identified the Regional Institute of Medical Sciences (RIMS), Imphal; Assam Medical College and Hospital (搜索) (AMCH), Dibrugarh; and the Institute of Post-Graduate Medical Education and Research (搜索) (IPGMER), Kolkata, as CoEs serving the eastern and northeastern regions.
The child's parents subsequently visited AMCH, Dibrugarh, where Manashree was registered and evaluated. On August 21, the CoE provided 12 vials of Risdiplam, which, according to the Health Department, are expected to cover approximately eight months of treatment. As per the prescription and treatment plan issued by the Dibrugarh CoE, the medication provided is valued at around Rs 2.29 lakh.
Because the medicine was supplied under the Rare Disease Programme, the family remains eligible for further assistance of up to Rs 47.31 lakh, subject to the provisions of the programme and recommendations of the concerned Rare Disease Committee.
The CoE has also reportedly informed the family that while Risdiplam has shown effectiveness in other forms of SMA, its efficacy in SMA Type 1 is very limited.
AIIMS Panel Backs Zolgensma, Pending Regulatory Approval
According to the Health Department, Manashree was evaluated by an expert team at AIIMS, New Delhi, in July. The AIIMS assessment concluded that the child may be considered for treatment with Zolgensma (Onasemnogene abeparvovec). The expert committee noted that the therapy replaces the deficient SMN1 (搜索) gene but cannot reverse motor-neuron degeneration that has already occurred.
The committee recommended Zolgensma with the objective of disease modification and preservation or improvement of residual motor function rather than normalisation of development. However, the assessment also noted that the drug was awaiting approval from the Drug Controller General of India (搜索) at the time of the evaluation.
Philanthropic Support and the Funding Arithmetic
The family had sought a certificate from the Tripura government regarding the financial assistance available for the child's treatment, which was required to approach Tata Trusts. The Medical Superintendent of Agartala Government Medical College and GB Pant Hospital issued the certificate on July 27. Based on the certificate, Tata Trusts has reportedly agreed to provide Rs 20 lakh towards Manashree's treatment.
The Tripura Health Department said government officials remain in regular contact with Manashree's parents and are coordinating to provide the best possible treatment and necessary assistance to the child.
Parallel Case Exposes Crowdfunding Verification Gap
A related case in the same district has drawn attention to the limits of India's rare disease financing architecture. West Tripura Member of Parliament and former Chief Minister Biplab Kumar Deb appealed to Union Health and Family Welfare Minister JP Nadda to facilitate life-saving treatment for 22-month-old Baby Mansari Chowdhury of Hapania, West Tripura, also diagnosed with SMA Type-I.
In a letter dated July 18, 2026, Deb enclosed a representation submitted by the child's father, Dhruba Chowdhury, stating that doctors at AIIMS, New Delhi, have recommended Zolgensma Gene Therapy, regarded as one of the most advanced treatments available for SMA Type-I. The letter states that the estimated cost of the treatment is around Rs 16–18 crore, an amount far beyond the financial capacity of the family. The MP requested the Union Health Minister to issue necessary directions to facilitate the therapy on humanitarian grounds.
The case highlights a structural limitation of the NPRD 2021: while patients with notified rare diseases, including SMA, are eligible for financial assistance of up to Rs 50 lakh at designated Centres of Excellence, the policy also acknowledges that certain advanced therapies, including gene therapies such as Zolgensma, cost several crores of rupees and therefore require additional financial support beyond the government's assistance.
To address this challenge, the Ministry of Health and Family Welfare has established the Government of India Digital Portal for Crowdfunding and Voluntary Donations for Patients with Rare Diseases. The portal enables verified patients, whose details are uploaded by their treating Centres of Excellence, to receive donations directly through an official and transparent government mechanism. Individual donors, corporate organisations and philanthropic institutions can contribute securely through the portal, with donations going directly to the treating institution for the patient's care. If donations exceed the required amount, the surplus is utilised for the treatment of other rare disease patients.
A search of the official Government of India Rare Diseases Crowdfunding Portal as of July 19, 2026, however, did not show any publicly listed crowdfunding appeal for Baby Mansari Chowdhury of Tripura. While the portal contains details of thousands of registered patients from across the country, including several SMA patients, no official listing for Mansari Chowdhury could be verified at the time of publication. Some individuals in Tripura are attempting to generate funds through crowdfunding by their own means.
This suggests the child's case may still be undergoing documentation or registration by the treating Centre of Excellence AIIMS, or that the official fundraising page has not yet been made publicly available. Until such registration is completed, there is no publicly verifiable Government of India crowdfunding appeal for the child.
The Tripura Government has already extended administrative support to the family following the intervention of Chief Minister Prof. (Dr.) Manik Saha. Officials of Tripura Bhawan in New Delhi have been coordinating with AIIMS to facilitate medical evaluation and treatment for the child. The issue has also generated widespread public sympathy, with several individuals and organisations expressing their willingness to support the family through various initiatives.
Experts in Tripura say that, in view of the Government of India's established policy for funding treatment of rare diseases, any large-scale public financial contribution should ideally be channelled through the official Ministry of Health and Family Welfare Rare Diseases Crowdfunding Portal once the patient's case is formally registered. Such a mechanism ensures transparency, institutional oversight, proper utilisation of donations and direct transfer of funds to the treating institution, while protecting donors and beneficiaries alike.