Families in Goa are anxiously awaiting the promised free medication for their children battling spinal muscular atrophy (SMA), a devastating genetic disorder. Ruby Borges, mother of nine-year-old Dylan, observes her son’s condition steadily worsening. She clings to the state government’s commitment from 2024 to provide costly medication at no charge. Despite a recent announcement by Health Minister Vishwajit Rane about a new pricing policy for life-saving therapies, these families continue to wait, hoping for relief.
The Impact of Spinal Muscular Atrophy on Children
Dylan previously received a year’s supply of medication in 2021 after his mother tirelessly crowdfunded and solicited donations. This effort, which involved going door-to-door across Goa, raised approximately Rs 45 lakh. Consequently, Dylan’s health significantly improved during 2021-2022. He regained the ability to sit unsupported, hold his neck straight, and grasp objects. However, after the medication ran out, his muscles weakened. Now, Dylan experiences frequent pneumonia, leading to prolonged intensive care unit stays. His mother explains that his hand pains when writing, and he struggles to sit comfortably in his wheelchair.
Spinal muscular atrophy is a severe neuromuscular disease impacting motor neurons, causing progressive muscle weakness. Globally, it is a leading genetic cause of death among children. Treatments, such as Risdiplam, can slow disease progression and enhance muscle strength. Dr. Philomena D’Souza, director of Novi Survat, actively pursues financial assistance from the Goa government for a consistent supply of Risdiplam for four children, emphasizing the urgent need for treatment to prevent further deterioration.
Challenges of Accessing SMA Treatment in India
Accessing SMA treatment in India presents significant financial hurdles. Risdiplam, approved by the CDSCO and DCGI in India, is one of only three global treatments for SMA and the sole one approved within the country. A bottle of Roche’s brand-name Risdiplam, marketed as Evrysdi, costs approximately Rs 6.2 lakh. Conversely, Natco Pharma launched a generic version at a considerably lower price of Rs 15,900 per bottle in April 2025. This vast price difference underscores the struggle for affordability. Furthermore, the Supreme Court of India has questioned why Risdiplam costs significantly more in India compared to neighboring countries like Pakistan and China, seeking explanations from the Centre and Roche.
The Indian government has introduced initiatives to address rare diseases. The National Policy for Rare Diseases (NPRD) 2021 offers financial support up to Rs 50 lakh for treatment at designated Centres of Excellence. Moreover, the government provides full waivers of Basic Customs Duty (BCD) and Integrated Goods and Services Tax (IGST) for imported drugs intended for personal use in SMA treatment. Nevertheless, the Centre informed the Supreme Court that providing free gene therapy for all 3,500 estimated SMA patients nationwide could cost an unsustainable Rs 35-40,000 crore annually.
Community and State-Level Initiatives for Spinal Muscular Atrophy Care
Several organizations and state governments are working to improve SMA care and access to treatment. Novi Survat Goa, a children’s palliative care organization, established the SMA Care Group in Goa in 2023. This group offers a platform for parent support, mutual encouragement, and shared experiences. Similarly, the Cure SMA Foundation of India, a prominent parent-led advocacy group, has been crucial in the launch of Risdiplam in India and continues advocating for its widespread accessibility. They welcomed the allocation of over Rs 900 crore by the Ministry of Health and the Delhi High Court for rare disease treatments.
Some states have also taken proactive steps. Karnataka, for instance, launched free SMA treatment at the Indira Gandhi Institute of Child Health in Bengaluru, supporting 50 children annually. Kerala became the first state to provide free Risdiplam, distributing the drug to 14 children. These regional efforts demonstrate a commitment to easing the burden on families and improving patient outcomes, even as a broader national solution remains elusive for many.
Looking Ahead: Hope for SMA Patients
While the wait for promised government assistance continues for families like Dylan’s in Goa, the growing awareness and collective efforts offer hope. The introduction of more affordable generic options like Natco Pharma’s Risdiplam, coupled with ongoing advocacy by organizations such as Novi Survat and Cure SMA, plays a vital role. Consistent government intervention, along with a focus on accessible and sustainable treatment models, will be crucial in providing a better quality of life for all children affected by spinal muscular atrophy in India.
Frequently Asked Questions
Q1: What is Spinal Muscular Atrophy (SMA)?
Spinal Muscular Atrophy (SMA) is a rare genetic disorder affecting motor neurons in the spinal cord. It leads to progressive muscle weakness, impacting movement, swallowing, and breathing. It is a leading genetic cause of death among children globally.
Q2: Is treatment for SMA available in India, and how much does it cost?
Yes, Risdiplam is currently the only SMA treatment approved in India. While Roche’s brand name version (Evrysdi) costs around Rs 6.2 lakh per bottle, a generic version launched by Natco Pharma is significantly more affordable at approximately Rs 15,900 per bottle. The overall treatment cost varies based on dosage and duration.
Q3: What initiatives has the Indian government taken to help SMA patients?
The Indian government’s National Policy for Rare Diseases (NPRD) 2021 provides financial support up to Rs 50 lakh for treatment at designated Centres of Excellence. Additionally, imported drugs for personal use in SMA treatment receive waivers for Basic Customs Duty (BCD) and Integrated Goods and Services Tax (IGST).
References
- Families wait for promised medication for kids with spinal muscular atrophy – ETHealthworld
- Risdiplam India – Cost, Uses, Approval & Why It Is Expensive – Impact Guru
- India Launches Risdiplam at ₹15K: Major Relief for SMA Patients – MrMed
- Free SMA treatment for all to cost Rs 40000 crore a year, not SC’s call: Government – The Times of India
- SC to govt: Why can’t SMA med be as cheap in India as in Pakistan, China? – The Times of India
- Natco to price Risdiplam generic at ₹15,900 – The Hindu
- Spinal Muscular Atrophy: Indira Gandhi Institute to offer free treatment for children – The Economic Times
- Drugs for SMA given to 14 children – Organization for Rare Diseases India
- Initiatives by the Government for treatment of rare diseases – PIB
- Cure SMA Foundation acknowledges budget allocation for rare disease treatments – The Economic Times
- The Goan EveryDay: Promise of ‘Novi Survat’ for kids with SMA
- Cure SMA Care Center Network – Cure SMA
- CureSMA – CureSMA India
Disclaimer: This article was automatically generated from publicly available sources and is provided for informational and educational purposes only. OC Academy does not exercise editorial control or claim authorship over this content. It is not a substitute for professional medical advice, diagnosis, or treatment. Always consult a qualified healthcare provider and refer to current local and national clinical guidelines.