Crowd gathers on Indore streets to fundraise for sick child:3-year-old girl battling rare disease, needs ₹9 cr injection from US

Indore’s 3-year-old Kanika is battling Spinal Muscular Atrophy (SMA) Type-2, a rare disease with one of the world’s most expensive treatments. The injection required for her treatment costs around Rs 9 crore. Her parents, Praveen and Sangeeta Sharma of Dwarkapuri, have been reaching out to actors, political leaders, ministers, and officials to arrange funds for her treatment. Fund raised for treatment Dainik Bhaskar learned from experts what this disease is and why the injection used in its treatment is so expensive. What is the disease, and how dangerous is it?
According to Dr. Hemant Jain, a paediatrician from Indore and former Dean of MGM Medical College, in the rare neuro-muscular genetic disease Spinal Muscular Atrophy (SMA), the child’s muscles gradually weaken. Signals from the brain and spinal cord to the muscles decrease as the spinal motor nerve cells get damaged. The brain cannot send messages to move the muscles, causing loss of body control. Initially, weakness affects the hands, feet, and body. Over time, it may become difficult for the child to sit, walk, breathe, or swallow. In severe cases, SMA can lead to paralysis or even death. It ranges from Type 1 to Type 4, affecting people of different ages. Disease can be identified in mother’s womb itself
Dr. Hemant Jain stated, This disease can be identified when the child is in the mother’s womb. Many children are unable to move properly in the womb. In such cases, specialist doctors test their cells. That’s when the disease is detected. If this happens, as soon as the child is born, if he receives timely treatment, he can become completely healthy. The later this disease is detected, the more cells are destroyed over time, which cannot be recovered. Even the injections available for this cannot revive dead cells. New cells can be prevented from being affected by it. Dr. Jain further stated that if the woman becomes pregnant again, she should undergo genetic counseling. A tissue sample is taken from the baby to perform an amniocentesis (gene) test. This can detect SMA disease in the unborn child. Finding patients for trials is also difficult
Dr. Jain said that any drug is trialed before being brought to the market. In India, one patient out of every 10,000 people suffers from SMA. Therefore, it becomes very difficult to find patients for its trials and studies. Stating the second reason, Dr. Jain said that SMA disease is treated with gene therapy. This means that a new or altered gene is inserted into cells to cure a disease, so that they can make healthy proteins or correct defective genes, which is why this medicine is also expensive. Zolgensma injection offers hope for SMA children According to media reports, the Zolgensma injection replaces the defective gene causing Spinal Muscular Atrophy (SMA) with a new gene, which becomes part of the child’s DNA. Once incorporated, the disease does not recur. In India, over 90 children have received the injection, although it is not considered a guaranteed cure. Experts say that if administered at the right time, the child can become completely healthy. What is Zolgensma?
Zolgensma, developed for the treatment of Spinal Muscular Atrophy, is a gene-therapy-based drug. It delivers a copy of a healthy SMN gene into the body. This allows motor neuron cells that control muscles to function properly.