Cure SMA Foundation of India, the leading organization driven by parents advocating for spinal muscular atrophy (SMA) patients across the nation, expresses gratitude for the significant advancements by India’s government, judiciary, and the broader healthcare sector in tackling rare diseases.
In a groundbreaking and unparalleled initiative, the Union Ministry of Health & Family Welfare alongside the Delhi High Court has allocated over Rs. 900 crore towards rare diseases, including SMA, to kickstart centralized procurement processes for rare disease treatments.
These historic undertakings signify a firm commitment from the Indian government toward the rare disease fraternity. Our heartfelt thanks go to Prime Minister Narendra Modi, Union Health Minister J.P. Nadda, and Justice Pratibha Sinha from the Delhi High Court for this development.
CureSMA emphasizes the need for the government to promptly commence the procurement process as mandated by the court within a 45-day timeframe and pledges their assistance to governmental bodies to accelerate proceedings.
SMA is a critical, progressive genetic neuromuscular disorder leading to the loss of vital physical abilities such as mobility, nourishment, and breathing. It is the predominant genetic cause of mortality among children globally. Although internationally approved treatments like Spinraza (Nusinersen), Zolgensma, and Risdiplam have revolutionized SMA care, access in India was elusive until the Drug Controller General of India (DCGI) sanctioned Risdiplam in 2020. This development has filled the SMA community with optimism and countless Indian children have since benefited from this vital therapy.
Alpana Sharma, Founding Director of Patient Advocacy at CureSMA, Mumbai, states, “Annually, around 4,000 children in India are born with SMA, making it a significant yet rare health challenge. The disorder is attributed to alterations in the SMN1 gene, resulting in severe muscular weakness, impacting crucial activities like movement, swallowing, and breathing. Among the three treatments available globally—Zolgensma, Spinraza, and Evrysdi (Risdiplam)—only Risdiplam has received approval in India. We are grateful for the availability of this vital medicine within our country, as it provides a new life opportunity for SMA patients. Collaboration among the government, pharmaceutical industry, and patient groups is essential to establishing a sustainable access framework, ensuring broader treatment reach nationwide. We have strong hopes for seeing such a sustainable solution achieved.”
Archana Panda, Founding Director of Patient Advocacy at CureSMA, Gurugram, declared, “Securing SMA treatment within India has posed a substantial challenge, but progress is underway due to collaborative efforts among the judiciary, government, pharmaceutical industry, and patient organizations. With the new budgetary allocation for rare diseases in India, this represents a new hope for SMA patients. As SMA progresses, any delay hinders our warriors’ rights to health. The life-altering medications can halt disease advancement and preserve functions in SMA patients. Collaborative efforts to quickly implement policies and ensure all patients access life-saving treatments are imperative.”
Moumita Ghosh, Founding Director of Event and Family Support at CureSMA, Kolkata, stressed that managing SMA extends beyond straightforward treatment—it necessitates a holistic, multidisciplinary approach. From physical rehabilitation to psychological counseling, all components contribute to enhancing patient outcomes. Constructing a sustainable ecosystem for SMA in India requires focusing on empowering healthcare professionals, bolstering patient support mechanisms, and fostering a collaborative specialist network. This integrated care model is crucial for ensuring long-term benefits for patients and their families.
Dr. Razeena Sethunat, Founding Director of Task Force and Patient Empowerment at CureSMA, Kerala, stated, “Kerala stands as an exemplary model of SMA patient support through pioneering funding solutions and comprehensive care. By utilizing state-level programs like the NPRD fund, Kerala has facilitated access to life-saving therapies and multidisciplinary care for over 100 SMA patients, including adults—a first in India. The state’s initiatives, such as the KARE program (Kerala United Against Rare Diseases), demonstrate the transformative potential of targeted funding and focused care for improving lives and offering optimism to families. National-level programs similar to the NPRD fund, when implemented efficiently, could replicate such success throughout the country, ensuring timely treatment and care accessibility for all rare disease patients.”
Srilakshmi Nalam, Founding Director of Patient Advocacy at CureSMA, Hyderabad, emphasized that establishing efficacy through robust data is pivotal in making informed decisions and building trust among patients and caregivers. Our efforts should prioritize accessible, effective, and evidence-based solutions. We express our gratitude to the Honourable Prime Minister and the administration for their dedication to rare disease care. The establishment of the Anusandhan National Research Foundation under the DST exemplifies a forward-thinking approach towards health solutions for conditions like SMA. Such initiatives offer hope and are fundamental in constructing a more promising future for the rare disease community.”
Key discussion points included: Recognizing breakthroughs and the importance of early intervention in the Current Treatment Landscape, acknowledging support from the government and judiciary while urging immediate policy implementations under Collaborative Efforts, establishing protocols for multi-disciplinary care, assistive technologies and inclusive policies within the Sustainable Ecosystem, noting Kerala’s success in providing comprehensive care for SMA under State-Level Models, along with CureSMA Foundation’s efforts in promoting policy changes, advancing MDT models, and encouraging research.