Hungarian toddler Zente treated with world's most expensive drug after fundraising campaign

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By Luke Hurst
Zente has now started treatment with access to the world's most expensive drug
Zente has now started treatment with access to the world's most expensive drug   -  Copyright  Zente SMA 1 Baby/Facebook

A poorly toddler has begun treatment that could save his life, after a crowdfunding campaign successfully raised more than €2 million to get access to the world’s most expensive drug.

Zente, who is 19 months old, needed to begin the treatment before he turned two, as the drug must be administered before that age to be effective.

His parents set up the crowdfunding campaign in Hungary to get him the treatment for a rare muscle disorder called Spinal Muscular Atrophy (SMA) - treatment which they labelled a "cure".

The drug, Zolgensma, costs €2.2m for one treatment.

Last month, six days after the campaign was started, Zente’s mother announced they had raised enough money for the treatment.

And now, that treatment has finally started.

A post on his Facebook campaign page thanked everybody for their support.

"I am happy to announce that zen received the you therapy treatment in Budapest today at the children's hospital! A terrible hard time is now over, and now a very important chapter begins.

"Zen is doing well right now, but strict quarantine, monitoring, and follow-up. I hope everything will be fine here too."

The medication uses gene therapy, is developed in the US, and is not yet registered either in Hungary or the EU.

In a clinical trial of the drug, 36 children with SMA received the treatment before they were two and by the end of the trial, none of them needed a permanent ventilation system to breathe.

Hungarian authorities issued a special permit for the use of the drug in Zente’s case.

SMA is a group of neuromuscular disorders that result in the loss of motor neurons and progressive muscle wasting.