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Leading medical professionals specializing in Spinal Muscular Atrophy (SMA) have called on the Indian central government to significantly enhance policies and funding dedicated to supporting SMA patients. This urgent appeal was made during the inaugural International Conference on Neuromuscular Disorders, APND 2025, which commenced on Friday.
Drs. Mary Iype and K.P. Vinayan, prominent figures in the field, highlighted the devastating impact of SMA, a progressive neuromuscular disease characterized by muscle weakness, mobility loss, respiratory complications, and potentially fatal outcomes.
“While global advancements in gene therapy and targeted treatments have drastically improved patient outcomes, access to these therapies in India remains a significant hurdle due to exorbitant costs and infrastructural limitations,” they stated.
They acknowledged the National Policy for Rare Diseases (NPRD 2021) as a positive step, offering financial aid of up to Rs 50 lakh for specific treatments and establishing Centres of Excellence (CoEs) for improved diagnosis and care. However, they stressed that “many patients continue to face barriers in accessing timely treatment due to funding delays, high drug prices, and limited availability of specialised care. The judiciary has intervened in several cases to ensure that funds promised for CoEs are released promptly, highlighting the gaps in policy execution. Given the high mortality associated with neuromuscular disorders, delays in treatment access amount to a denial of justice for affected children.”
The experts advocated for bolstering the national approach to rare diseases through preventive strategies, including genetic counseling and family tree mapping, to mitigate the burden of these disorders. They also emphasized the need for incentives to encourage domestic production of orphan drugs—specialized medications for rare diseases.
“In developed countries, governments have introduced schemes to promote investment in orphan drug research and manufacturing. India, they argue, must adopt similar measures and negotiate directly with pharmaceutical innovators to reduce drug costs and improve accessibility,” Dr. Iype explained.
Kerala has emerged as a leader in rare disease management through its KARE (Kerala United Against Rare Diseases) initiative, providing free treatment and multidisciplinary care for affected children. Notably, Kerala is the only Indian state to successfully offer free drug therapy to over 100 SMA patients, setting a national benchmark.
Dr. Giovani Baranallo, Professor at University College London, praised Kerala’s comprehensive care model, which integrates early screening, genetic diagnosis, physiotherapy, nutrition, and respiratory support.
Experts at APND 2025 are hopeful that the conference discussions will catalyze stronger national policies, improved healthcare infrastructure, and increased financial support, ensuring timely and affordable care for SMA and other rare disease patients.
Disclaimer: This news article is based on information provided and should not be considered medical advice. Readers are advised to consult with healthcare professionals for any health concerns. The financial information and policies discussed are subject to change and should be verified with official sources.
