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.