Mirk\u00f3 was born in 2015. He started life as a healthy-looking, happy little boy, and today he is a smiling nine-year-old child who faces an extremely serious disease with amazing courage: Duchenne muscular dystrophy.
Her family was diagnosed three years ago and since then they have been working hard to give Mirko the best possible future.<\/p>\n
Duchenne muscular dystrophy is a progressive, incurable genetic disease that causes the gradual deterioration of muscles. Mirko has a defect in exon 55, which first affects the skeletal muscles and later the respiratory and cardiac muscles. Doctors predict that by the age of ten to eleven he could be confined to a wheelchair and by his mid-teens he will need a respiratory support. The outcome is cruel: many people do not live into their twenties.<\/p>\n
The little boy is on steroid treatment, which, although it slows down muscle atrophy, also has serious side effects - including osteoporosis and an increased risk of bone fractures. Mirk\u00f3 regularly attends physiotherapy, swimming and rehabilitation to keep him mobile for as long as possible. His family has been trying to gain access to gene therapies and exonskiping treatments for two years now, but so far the doors of all research programmes have remained closed to them.<\/p>\n
Now, however, hope has finally dawned. Mirk\u00f3 has received approval for a gene therapy treatment called Elevidys, which could be a breakthrough in the treatment of the disease. The only - but huge - obstacle is the price: HUF 1.1 billion. This sum is far beyond the means of one family. Moreover, time is short: the therapy is only available to children who are able to walk, and in Mirko's case it is only a matter of months.<\/p>\n
Together, let's help Mirko to make his laughter the loudest around him, not his illness.<\/p>","protected":false},"excerpt":{"rendered":"
Mirk\u00f3 was born in 2015. He started life as a healthy-looking, happy little boy, and today he is a smiling nine-year-old child who faces an extremely serious disease with amazing courage: Duchenne muscular dystrophy.
\nHer family was diagnosed three years ago and since then they have been working hard to give Mirko the best possible future.<\/p>","protected":false},"author":1,"featured_media":2880,"comment_status":"closed","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[40],"tags":[],"class_list":["post-2877","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-orokbefogadott-csaladok"],"yoast_head":"\n