Following the successful fundraising for Zente and Levente, two Hungarian toddlers suffering from SMA (spinal muscular atrophy) for their 700 million HUF treatment, the most expensive medicine in the world, Hungary has come together again for a third boy suffering from SMA – Noel, who lives in Érkörtvélyes in Bihar county, Romania.
Noel’s mother reported on a Facebook page created for the baby, who is only a few months old, is suffering from the same illness as the two other boys who recently received the support of a whole country, which made it possible for them to receive treatment. The family has created a foundation, but people can donate to multiple bank accounts as well.
Fundraiser Set up for Another Toddler to Receive World’s Most Expensive Medicine
The mother emphasized that she “would like to point out that I not only ask Hungarian people and those living in Hungary to help. We created the site in Hungarian because it is our mother tongue and because there are many helpful Hungarian people living here in Romania as well. Of course, translations are being made.”
They have already taken the necessary steps and are waiting for an approval for Noel to receive another medicine, Spinraza, which will help him to develop, and for his condition to not further deteriorate. The Spinraza injection is financed completely by the National Health Insurance Fund (NEAK). The vaccine was patented just last year. This treatment is not cheap either, as an injection costs 23 million HUF, but the cost is entirely borne by NEAK. However, this medicine is needed by patients for the rest of their lives.
Fundraising for Toddler’s Expensive Treatment Moves Hungary
This is why they have also set up fundraising to receive a medicine which is only needed once to improve the baby’s condition. However, this expensive medicine, called Zolgensma, is currently the most expensive medicine in the world and it has only been on the market since May.
SMA-1 is an extremely rare genetic disorder which affects only one in eight to ten thousand people. Because of a defective gene, their body does not produce the protein that protects muscle cells, so their muscles slowly deteriorate. Symptoms of SMA-1 usually occur during the first months of the patient’s life. In most cases, due to respiratory paralysis, children do not reach age two. There are approximately 120 SMA patients officially registered in Hungary (this applies to all types of SMA, not just SMA-1) but due to the outdated registration system, professionals say that the actual number is around 300.
The essence of the treatment is that the patients are given a virus by gene therapy that “infects” and replaces the gene pool of defective or missing motor neurons, thus preventing muscular atrophy. In Hungary, another treatment may have given hope to SMA patients as of last year, but for the time being, it is only funded on a case-by-case basis, exclusively for children.
featured photo: Noel and his family. (photo: Kicsi Noel – SMA 1 Facebook)