What is spinal muscular atrophy and why does it require an Dh8m treatment?

Published by: UAE
Written by: Gillian Duncan

Spinal muscular atrophy (SMA) is a debilitating condition that affects around one in every 10,000 children.

Zolgensma, the most effective treatment, is also the world’s most expensive drug.

A single one-time infusion of the drug costs Dh8 million.

But why does it cost so much?

The National explains.

What is SMA?

SMA is a hereditary disease caused by a missing or faulty gene that the body requires to make a protein essential for motor neuron cell survival.

Without sufficient levels of the protein, the motor neurons – which are nerve cells in the brain stem and spinal cord that control activities such as speaking, walking, breathing, and swallowing – die, leading to muscle weakness and atrophy. Click here to read the rest of the story.

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