A Turkish family from Leicester have made a desperate appeal for help as they seek to raise nearly two million pounds to fund lifesaving treatment for their toddler son Metehan Fidan.
Earlier this year, Metehan was diagnosed with type 1 spinal muscular atrophy (SMA). This rare neuromuscular disease severely debilitates the body: babies with the condition have very weak and floppy arms and legs, and they have problems moving, eating, breathing, and swallowing. They are also unable to raise their heads or sit without support.
The life expectancy for children with type 1 SMA is very short, with many dying in the first few years of life due to breathing difficulties.
Parents Tuncay and Zeliha Fidan are desperate to save their only child and help may be at hand if they can raise enough money to take their son to the USA to receive pioneering treatment.
Father Tuncay told T-VINE: “Due to a missing gene in Metehan, he can’t use his muscles, which means he can’t move, can’t sit, eat, or most importantly breathe.
“The treatment of SMA patients is available with the medication Zolgensma, but the price of this medication is exactly $2.4 million and can only be treated in the USA. Unfortunately, this treatment is not funded by NHS.”
Although it comes with a huge price tag Zolgensma is the only one-dose option for SMA sufferers and clinical trials have shown it to be highly effective. The drug is given as an intravenous infusion, which makes it easier to administer to SMA patients.
“I do not want my baby to die from this illness knowing there is a treatment for it”
The only other alternative on the market is Spinraza, which requires multiple spinal infusions. Although the costs of this drug is initially cheaper – $750,000 for four doses in year one – Spinraza works out much more expensive in the long-run, as the treatment must continue for life and each year’s injections cost $375,000. This drug also poses more pain for patients and additional risks through ongoing lumbar punctures.
Zolgensma may become approved for the UK sometime in 2021, but that would be too late for Metehan.
In September, the Fidan family launched a fundraising campaign and have managed to raise over a quarter of a million pounds to date, with support coming from all over the UK and abroad. Footballer Mesut Özil and singer Yusuf Güney are among those to have helped raise awareness of Metehan’s desperate plight.
Fundraising efforts large and small continue, but time is not on the family’s side due to the ravaging effects of the disease on Metehan and also the criteria laid down by Novartis, the makers of Zolgensma.
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The pharmaceutical company says in order for the drug to be administered and be effective, a child must be younger than two years old and weigh under 13kg.
Metehan will turn one on 12 November 2020.
“As parents, we are fighting against time to get his medicine before the age of 2. Metehan does not have much time left. It hurts me to say, but I do not want my baby to die from this illness knowing there is a treatment for it,” Mr Fidan said.
Go Fund Me: https://www.gofundme.com/f/metehan039s-sma-type-1-treatment