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What’s SMA disease that is affecting kids and why is treatment so expensive?

Published: 1st Jul 2021 4:16 pm

Ayaansh Gupta is just 3-years-old. But he became one of the first patients in Hyderabad to be administered Zolgensma, a gene therapy with a list price of Rs. 16 crore. He was suffering from Spinal Muscular Atrophy (SMA). A similar case had recently come up in the city wherein the parents had resorted to crowdfunding.

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Here’s everything you need to know about SMA and why is the cure so expensive?

What is SMA?

Spinal muscular atrophy is a genetic disease affecting the part of the nervous system that controls our ability to move our muscles voluntarily. The brain stops sending messages that control muscle movement. It is usually spotted when babies are about three months old. It is the most common genetic cause of death in childhood. If untreated, the child rarely lives beyond two.

What causes SMA?

SMA is a disease that’s passed down through families. If your child has SMA, it’s because they have one copy of the missing gene from each parent. If the child gets a faulty gene from just one of the parents, they won’t get SMA but will be a carrier of the disease. When they grow up, they could pass the broken gene to their child.

What are common signs of SMA?

The child starts failing to meet the usual milestones like holding their head up or rolling over 0r kicking their legs. They experience trouble breathing. Where they are untreated, risk further damage to the nerve fibres and can make the difference between a child being able to walk or run or sit unaided.

Why is the treatment so expensive?

There are not many patients who have this disorder. In the last few years, at least three new treatment options for SMA have been approved. One of these is Zolgensma, made by Novartis, the second most effective drug for the disorder. The expertise required for making it and the research around it is said to be very long. Highly advanced infrastructure is required to make this single-dose therapy. It’s also a one-time dose and life-saving. The fewer the cases the higher its price will be, experts say.

To import Zolgensma from the USA or the European countries, one has to pay import duty and GST on the medicine.

How will Zolgensma treat SMA?

According to the United Kingdom National Health Service, the drug contains a replica of the missing gene. The active ingredient onasemnogene abeparvovec passes into the nerves and restores the gene, which then produces proteins necessary for nerve function and controlling muscle movement. The dose is determined based on the weight of the patient. It is a one-time intravenous infusion that is administered to a patient for over an hour.

What are the types of SMA?

Type 0 is the rarest and most severe form of SMA and develops while the mother is still pregnant. Babies with this type of SMA move less in the womb and are born with joint problems, weak muscle tone, and weak muscles for breathing. They often do not survive due to breathing problems.

Type 1 SMA’s biggest concern is weakness in the muscles that control breathing. Most children with type 1 SMA don’t live past age 2 because of breathing problem.


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