The Cure SMA Foundation of India, the nation’s largest parent-led advocacy group for Spinal Muscular Atrophy (SMA), has expressed its appreciation for significant steps taken by the Government of India, the judiciary, and the broader healthcare ecosystem to address rare diseases, including SMA.

In an unprecedented move, the Ministry of Health & Family Welfare, along with the Delhi High Court, has allocated over ₹900 crore for the centralized procurement of treatments for rare diseases, marking a historic turning point in India’s approach to rare disease care.

The decision reflects a strong and progressive commitment by the Indian government to the rare disease community, and the Cure SMA Foundation has expressed its gratitude to Prime Minister Shri Narendra Modi, Union Minister of Health & Family Welfare J.P. Nadda, and Justice Pratibha Sinha of the Delhi High Court. The Foundation has also urged the government to expedite the procurement process as directed by the court within 45 days and has pledged support to assist in speeding up the process.

Spinal Muscular Atrophy (SMA) is a severe, progressive, genetic neuromuscular disease that causes muscle weakness, affecting key functions such as walking, eating, and breathing. It is the leading genetic cause of death among children worldwide. In India, approximately 4,000 children are born with SMA each year, making it a critical, albeit rare, health challenge. Despite the global availability of treatments like Spinraza (Nusinersen), Zolgensma, and Risdiplam, access to these life-saving drugs remained a distant hope for many Indian families.

However, the approval of Risdiplam by the Drug Controller General of India (DCGI) in 2020 has brought new hope to the SMA community. Many patients have benefited from this treatment, which has transformed the outlook for SMA patients woworldwide.S

Alpana Sharma, a key advocate for SMA patients, emphasized the importance of collaboration between the government, the pharmaceutical industry, and patient groups. “While Risdiplam is now available in India, more needs to be done to ensure that all SMA patients have access to treatment. We need to work together to build a sustainable access network so that more patients can benefit from this life-saving drug,” Sharma stated.

Archana Panda, another SMA advocate, highlighted the urgency of providing timely treatment to SMA patients. “Every day of delay in accessing life-saving medicines means more deterioration for SMA patients. These treatments can halt disease progression and preserve essential functions. We must expedite the implementation of government policies to ensure timely access to treatments,” she said.

Moumita Ghosh stressed that managing SMA requires a multi-disciplinary approach beyond just treatment. “Physical therapy, psychological support, and specialized care play a crucial role in improving the quality of life for SMA patients,” Ghosh said. She added that creating a comprehensive network of specialists and healthcare providers is essential to ensure the long-term well-being of SMA patients in India.

Razeena, a prominent advocate from Kerala, highlighted the state’s success in providing comprehensive care for SMA patients. Kerala’s NPRD fund and the KARE program (Kerala United Against Rare Diseases) have provided innovative funding solutions and access to life-saving therapies for over 100 SMA patients, including adults.

Razeena noted that the Kerala model could serve as an inspiration for national-level programs like the NRDP fund, which could ensure timely access to treatment for all rare disease patients across India.

Srilakshmi Nalam expressed her gratitude to the government for its commitment to rare disease care, particularly SMA. “The establishment of the Anusandhan National Research Foundation under the DST is a visionary step towards advancing healthcare solutions for SMA and other rare diseases,” Nalam said. She emphasized the importance of evidence-backed, accessible, and effective solutions for the rare disease community in India.

The historic measures taken by the Government of India and the judiciary mark a new era for the rare disease community, offering hope for SMA patients and their families across the country.

The Cure SMA Foundation continues to work tirelessly to ensure that these policy changes translate into real-world improvements in access to care and treatment for SMA patients.