Cure SMA Foundation acknowledges budget allocation for rare disease treatments

Cure SMA Foundation of India, a parent-led patient advocacy organisation for Spinal Muscular Atrophy (SMA), has commended recent steps taken by the Government of India, the judiciary, and healthcare stakeholders to address rare diseases. The Ministry of Health and Family Welfare and the Delhi High Court have allocated over Rs 900 crore for rare disease treatment, including SMA, marking a milestone for the community. This initiative aims to centralise procurement of treatments for rare diseases.

Cure SMA expressed gratitude to the Prime Minister, the Health Minister, and the judiciary for their role in facilitating this progress. The foundation called for the immediate initiation of the procurement process, as directed by the court, within 45 days. It also pledged its support to expedite the process.

SMA is a genetic neuromuscular disease that severely affects physical abilities, including movement, swallowing, and breathing. It is the leading genetic cause of death among children globally. While treatments such as Spinraza, Zolgensma, and Risdiplam have transformed outcomes for patients worldwide, access in India has been limited. The approval of Risdiplam by the Drug Controller General of India (DCGI) in 2020 marked a turning point, offering new hope to Indian SMA patients.

SMA in India and the Current Treatment Landscape
Alpana Sharma, a representative of Cure SMA, emphasised the scope of the challenge in India, where approximately 4,000 children are born with SMA annually. She noted: “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.”

Collaborative Efforts for Treatment Access
Archana Panda highlighted the importance of collaboration, stating: “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 Organisation. 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.”

Holistic Care for SMA Management
Moumita Ghosh stressed the need for a multidisciplinary approach: “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.”

State-Level Successes as a Model
Razeena from Kerala shared insights into the state’s approach, noting: “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.”

Gratitude and Vision for the Future
Srilakshmi Nalam expressed appreciation for government initiatives and stressed the need for evidence-based solutions: “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. Such initiatives inspire hope and are instrumental in building a better future for the rare disease community.”

Cure SMA Foundation reiterated its commitment to advocating for policy changes, promoting multidisciplinary care models, and advancing research. The foundation also highlighted Kerala’s success as an example for national-level programmes to replicate. It underscored the urgency of implementing policies to ensure timely access to life-saving treatments for SMA patients across India.