Bengal boy with rare genetic disease receives Rs 16-crore life-saving medicine

Despite the limited availability of treatment for Spinal Muscular Atrophy (SMA) due to high costs, a boy from West Bengal was successfully administered this life-saving drug.

In Short

• SMA treatment in India is limited due to high costs
• Boy from West Bengal received Rs 16-core medicine
• Hospitals depend on crowdfunding for treatment

Treatment options for Spinal Muscular Atrophy (SMA), a rare genetic disease, are virtually nonexistent in India, primarily due to the excessive costs. Zolgensma, the drug needed for the gene therapy to treat this condition, is priced at an astounding 16 crore rupees. However, Din Muhammad from West Bengal managed to receive this life-saving medicine.

Din Muhammad was born into an ordinary family in East Medinipur. When he was six months old, his mother noticed that he struggled to move his arms and legs. He could barely lift his legs, and his hands moved very slowly. He also had difficulty lifting his head, sitting up, or crawling.

Initially, he was taken to a local paediatrician, but upon the doctor's recommendation, the family travelled to Kolkata. After extensive testing, he was diagnosed with Spinal Muscular Atrophy (SMA).

Ravindra Pai, Managing Director of Peerless Hospital, said, "SMA is quite a complex and challenging disease. Peerless has the necessary ecosystem to treat it. Doctors at the hospital also work very hard to treat this complex disease and get the best treatment for the affected children."

The physicians at the Peerless Paediatric Department lead this therapy. According to them, only three children in East India have received this treatment so far. Two of these children were treated at Peerless Hospital in Kolkata, and one at NRS Hospital in Kolkata. To date, three children have completed the necessary paperwork to be eligible for this therapy.

“This therapy is most effective when administered before the age of two,” says Dr Sanjukta De. “Three children, aged between 7 to 15 months, are currently in line to receive this treatment. We are working to raise funds for them through crowdfunding.”

Zolgensma (onasemnogene abeparvovec-xioi) is a groundbreaking gene therapy used to treat spinal muscular atrophy (SMA), a rare genetic disorder that results in the loss of motor neurons in the spinal cord, causing muscle weakness and atrophy. However, it is now considered less rare, with a worldwide prevalence estimated at 1 in 8,000.