Spinraza is currently the only approved medicines for the rare Spinal Muscular Atrophy; state government approves decision after two-year-old from Sion was recently diagnosed with it
A cream/ivory ribbon symbolises support for Spinal Muscular Atrophy among other similar genetic diseases. Representation pic/Getty Images
The civic-run KEM hospital is all set to provide free treatment to pediatric patients suffering from the rare genetic disorder called spinal muscular atrophy (SMA) type 1, 2 and 3. With this, it becomes the first civic-run hospital in Maharashtra to provide free treatment for the rare disease with Spinraza - currently the only approved drug for its treatment.
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The move came after a two-year-old from Sion was diagnosed with the rare disease when he was six months old. The type 1 SMA restricted his movement and growth with the voluntary movement of his muscles confined. He cannot walk or move like children his age can and thus has no control over his excretory system either. For pediatric patients suffering from this disease, the life expectancy is usually as short as two to three years but can vary to some extent.
Considering the high price of Spinraza, the decision to supply the medicine free-of-cost at KEM hospital was approved by the state government. "Spinraza is the only approved drug available across the globe for SMA treatment. Giving it free-of-cost to patients would certainly give them some hope of recovery," said Dr Hemant Deshmukh, dean of KEM hospital.
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According to the official website of the Spinraza company, around 2,600 patients with SMA — from a three-days-old to a 79-year-old — have been treated with the injection in the past two years, aiding their locomotive movement.
What is SMA?
It is a genetic disease that affects the nervous system of the body controlling the voluntary muscle movement. Research has shown that it is mainly caused by inadequate production of a protein called survival motor neuron (SMN) essential for motor functions. The nerve cells located in the spinal cord fail to send signals to muscles in the body which gradually stop its movement. This results in the patients remaining small in size since their muscles are not active.
Dr Deshmukh said that there are four types of SMA. "When an infant starts showing symptoms, especially in the lower part of the body, it is considered as type 1. Type 2 and type 3 are common among slightly elder kids. When an adult is diagnosed with the symptoms, they are classified into type 4," he explained.
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