Cure SMA Foundation of India, the largest parent-led patient advocacy organization for Spinal Muscular Atrophy (SMA) in the country, appreciates and acknowledges the positive strides made by the Government of India, the judiciary and the entire healthcare ecosystem towards addressing rare diseases in India. In a historic and unprecedented move, The Ministry of Health & Family Welfare and The Delhi High Court have earmarked ₹900+ crore for rare diseases, including SMA, for initiating centralized procurement of rare disease treatments. These historic measures reflect a strong and progressive commitment by the Government of India towards the rare disease community. We especially thank our Honorable PM Shri Narendra Modi ji, Union minister of Health & Family Welfare Honorable Sri J P Nadda and Justice Pratibha Sinha, Delhi High Court for this development.

CureSMA urges the Government to take necessary steps to initiate the procurement process as directed by the court within 45 days and pledges their support to help the government agencies in fastening the process.

SMA is a severe, progressive, genetic neuromuscular disease that robs individuals of essential physical abilities, such as walking, eating, and breathing and is the leading genetic cause of deaths among children worldwide. While globally approved treatments—Spinraza (Nusinersen) and Zolgensma and Risdiplam have transformed the outlook for SMA patients, accessibility in India remained an unfulfilled promise until the approval of Risdiplam by the Drug Controller General of India (DCGI) in 2020. This gave the SMA community in India hope, and today many Indian SMA patients have benefited from this life-saving drug.

Alpana Sharma – About SMA in India and Current Treatment Landscape

“In India, approximately 4,000 children are born with Spinal Muscular Atrophy (SMA) every year, making it a critical, albeit rare, health challenge. This genetic disorder, caused by a mutation in the SMN1 gene, leads to severe muscle weakness, impacting essential functions like movement, swallowing, and even breathing.

Among the three globally available treatments, Zolgensma, Spinraza (Nusinersen), and Evrysdi (Risdiplam)—only Risdiplam is approved for use in the country. We are thankful that this life-saving drug is available in the country, and is giving a new lease of life to SMA patients. All stakeholders including the government, pharma industry and patient groups need to work together to build a sustainable access network so that the treatment reaches more patients in the country. We are very hopeful that a sustainable solution is in sight.”

Archana Panda – Treatment Access Through Collaborative Efforts

“Ensuring access to treatment for SMA in India has been a monumental challenge, but progress is being made thanks to collaborative efforts between the judiciary, government, pharma industry and Patient Organization. Now with the new budget being announced for rare disease treatment in India, this is a new lease of life for SMA patients. SMA is a progressive disease, and every day of delay denies our warriors their right to good health. The life-saving medicines can not only arrest disease progression but also preserve functionalities in SMA patients. We must work together to expedite and implement the order as soon as possible and ensure that all patients get access to life saving medicine at the earliest.”Moumita Ghosh – Multi-Disciplinary Care and SMA Ecosystem

“SMA management extends beyond treatment—it demands a holistic and multi-disciplinary approach. From physical therapy to psychological support, every aspect of care contributes to improving patient outcomes. To build a sustainable ecosystem for SMA in India, we must focus on empowering healthcare providers, strengthening patient support systems, and creating a network of specialists who work in tandem. This integrated care model is vital for ensuring long-term benefits for patients and their families.”

Razeena – The Kerala Model of SMA Support

“Kerala has set an exemplary model in supporting Spinal Muscular Atrophy (SMA) patients through innovative funding solutions and comprehensive care. By leveraging state-level programs like the NPRD fund, Kerala has successfully provided access to life-saving therapies and multidisciplinary care for over 100 SMA patients, including adult patients—a first in India.

The state’s initiatives, such as the KARE program (Kerala United Against Rare Diseases), demonstrate how targeted funding and dedicated care can transform lives, offering hope to families and significantly improving patient outcomes. These impactful measures highlight the potential of national-level programs like the NRDP fund, which, if implemented swiftly, could replicate such successes across the country, ensuring timely access to treatment and care for all rare disease patients.”

Srilakshmi Nalam – Gratitude for Government Initiatives

“Establishing efficacy through credible data is critical to making informed decisions and fostering trust among patients and caregivers. Our focus should be on developing solutions that are accessible, effective, and evidence-backed. We are grateful to the Honourable Prime Minister and the government for their commitment to rare disease care. The establishment of the Anusandhan National Research Foundation under the DST reflects a visionary approach to advancing healthcare solutions for conditions like SMA. Such initiatives inspire hope and are instrumental in building a better future for the rare disease community.”

Key Discussion Points:

1. Current Treatment Landscape: Understanding breakthroughs and the importance of early intervention.
2. Collaborative Efforts: Recognizing government and judiciary support and urging immediate implementation of policies.
3. Sustainable Ecosystem: Establishing multidisciplinary care protocols, assistive technologies, and inclusive policies.
4. State-Level Models: Highlighting Kerala’s success in providing comprehensive care for SMA patients.
5. Cure SMA Foundation’s Role: Advocating policy changes, promoting MDT models, and advancing research.