Sick baby needs ‘world’s most expensive drug’
A COUPLE from North Cyprus have launched a desperate appeal to raise money for their sick baby to be treated with “the world’s most expensive drug”.
Five-month-old Karel, the second child of Şeyda and Yusuf Karaoğulları from Yeniboğaziçi, has been fighting for life for the last three months.
The baby boy was diagnosed with type 1 SMA (spinal muscular atrophy) and is currently being treated in the intensive care unit of a hospital in Ankara, the parents said.
Type 1 SMA, which develops in babies less than six months old, is the most severe form of the genetic condition that makes the muscles weaker and causes problems with movement, according to the UK’s NHS website.
The NHS says that in the past babies with type 1 rarely survive beyond the first few years
of life but in recent years outcomes have improved with early diagnosis and treatment.
It adds that there is no cure and that treatment and support is made available to manage the symptoms and help people with SMA have the best possible quality of life.
Little Karel’s parents said this week that they had obtained the “necessary permissions” to raise money for their son to be treated with a drug from the US called Zolgensma, which they say his survival depends on.
Zolgensma, manufactured by Novartis Gene Therapies, is a life-saving gene therapy drug that can help babies with the rare genetic disease move and walk – but has a reported list price of £1.79 million per dose.
In studies Zolgensma has helped babies to reach milestones such as “breathe without a ventilator, sit up on their own and crawl and walk after a single infusion treatment” according to the NHS.
The appeal for baby Karel has been spread on social media, with members of the public asked to donate as much as they can. Some have been raising funds by selling handmade items.
Writing on Facebook recently, Mrs Karaoğulları said: “Karel is currently in intensive care due to respiratory failure.
“Therefore, we are waiting for a price offer from the hospital in order to receive the gene therapy Zolgensma, which is the gene therapy in the USA, as he cannot receive the Spinraza [medicine] treatment that we started in Turkey because he is connected to a ventilator.”