Dubai: Hospital saves 69 lives with Dh10-million ‘wonder drug’ in less than 3 years

Dubai: Hospital saves 69 lives with Dh10-million ‘wonder drug’ in less than 3 years

Over time, the condition, which mostly affects infants and children, causes muscles to become weaker and waste away

Published: Sat 26 Aug 2023, 4:14 PM

Spinal muscular atrophy (SMA) is a rare genetic disease leading to the loss of nerve cells in the spinal cord called motor neurons, which control the movement of muscles.

Over time, the condition, which mostly affects infants and children, causes muscles to become weaker and waste away. And gradually it gets worse.

While there is no cure for this dreadful condition, the ray of hope comes in the form of gene replacement therapy. However, the lifesaving one-time dose comes with a price tag of around Dh8-10 million.

Back in November 2020, Dubai’s Medcare Women and Children Hospital became the first healthcare provider in the Mena region to offer free gene therapy to two infants suffering from SMA type 1 as part of the Global Managed Access Programme by Novartis.

Though the lottery system for complementary treatment ceased to exist, the hospital has been receiving a lot of patients, mainly from countries like Turkey, Romania, Russia, Belarus, Nepal, and Iran among others.

By the end of this month, the hospital has treated 69 children with gene replacement therapy, with three cases in August