As we reported a couple months ago, the whole country joined forces to raise 700 million forints for a toddler suffering from SMA (spinal muscular atrophy), as only the most expensive medicine in the world could save him. With the help of generous donations from the country and beyond, Zente could start the treatment in time, and now, three months after the expensive treatment, he finally stood on his feet.
Zente was diagnosed as a baby with the most severe type of muscular atrophy, SMA-1, an extremely rare genetic disorder which affects only one in eight to ten thousand people. Because of a defective gene, the body does not produce the protein that protects muscle cells, so muscles slowly deteriorate. The needed amount, 700 million forints was collected in less than a week for the expensive medicine called Zolgensma, which was good news for the toddler and parents, as they were also competing with time, as children are only allowed to receive the drug before reaching the age of two.
Now, three months after Zente received the treatment, his mother shared a video on Facebook, showing the toddler standing on his feet.
Zolgensma is currently the most expensive medicine in the world and it has only been on the market since May. The essence of the treatment is that 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. 36 infants aged between two weeks and eight months have participated in the clinical trials. According to statistics from September, all children receiving treatment showed improvements in their condition.
Fundraising Started for Another Baby with SMA to Receive World’s Most Expensive Medicine
There is another option for SMA patients, a medicine that is available in Europe. Back in December, Miklós Kásler, Minister of Human Capacities, announced that an agreement has been reached with the only drug distributor for the treatment of SMA, which is registered in Europe, so all children under the age of 18 will have access to the treatment.
featured photo: Zente’s Facebook page