Thiruvananthapuram, March 14 Leading medical professionals dealing with patients suffering from Spinal Muscular Atrophy (SMA) have urged the central government to strengthen policies and funding for such patients, emphasising the urgent need for better access to life-saving treatments.
This demand came at the first international conference on neuromuscular disorders, APND 2025, which opened here on Friday.
Leading medical professionals Dr. Mary Iype and Dr. K.P.Vinayan said SMA remains one of the most devastating neuromuscular diseases, causing progressive muscle weakness, loss of mobility, respiratory complications, and, in severe cases, early mortality.
“While global advancements in gene therapy and targeted treatments have significantly improved patient outcomes, access to these therapies in India remains a major challenge due to high costs and infrastructure limitations,” said the doctors.
They pointed out that India’s National Policy for Rare Diseases (NPRD 2021) was a step forward in addressing rare disease care, offering financial support of up to Rs 50 lakh for specific treatments and establishing Centres of Excellence (CoEs) to enhance diagnosis and care.
“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,” said the doctors.
The leading professionals through their expertise aim to strengthen the country’s approach to rare diseases through preventive strategies such as genetic counselling and family tree mapping, which can help reduce the burden of these disorders.
They also will advocate for incentives to encourage indigenous production of orphan drugs—specialised medications developed 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,” said Iype.
Incidentally over the years Kerala has emerged as a leader in rare disease management with its KARE (Kerala United Against Rare Diseases) initiative, which provides free treatment and multidisciplinary care for affected children.
Incidentally Kerala is the only state in India to successfully offer free drug therapy to over 100 SMA patients, setting a national benchmark in public healthcare.
Dr Giovani Baranallo, Professor at the University College of London, termed the way Kerala is going forward in SMA which integrates early screening, genetic diagnosis, physiotherapy, nutrition, and respiratory support, ensuring a comprehensive care model.
Experts are hopeful that the discussions will lead to stronger national policies, better healthcare infrastructure, and greater financial support to ensure that SMA and other rare disease patients receive the timely and affordable care they deserve.
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