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Today, it is a child who smiles like many others, but his story moved the world. Margaux was suffering from a very rare disease and therefore difficult to diagnose.
It is about Adenosine deaminase enzyme deficiency (Ada), a very rare autoimmune pathology that compromises so much the immune system that it is attacked without any defense against any type of pathogen. Those who suffer are forced to isolate themselves from the rest of the world and live in sterile environments. For this reason, young patients with this disease are called "bubble kids".
The history of Margaux
Margaux was born on September 20, 2013; continued to get sick, had a respiratory crisis, but no one understood the cause of the disease.
The first respiratory crisis occurred when the child was only 5 weeks old and at that time, the doctors had told the parents that the baby had nothing. Even the ensuing crises, although treated with antibiotics, did not create alarmism among doctors. Some pediatricians, says his father, said "he has something that he can not understand", While claiming that the baby was in good health"and sent him home".
The correct diagnosis takes place on 5 September 2014, after a very serious crisis caused by Margaux and his transfer to the University Hospital of Leuven, east of Brussels. The child is suffering from Ada. The doctors, who are evaluating the case, understand that the chances of healing with a stem cell transplant are not high. moreover, the little brother is not a compatible donor.
As a result, parents contacted Alessandro Aiuti, researcher of the foundation Telethon and medical researcher of the San Raffaele from Milan. The girl has therefore undergone an innovative treatment. The therapy is the result of a scientific experiment supported by European research funds.
Since 2015, the girl has progressed and today, she is completely healed and, like all the other children, she plays and goes to school. Today, Margaux celebrates two birthdays: birth and rebirth.
The emotion of the European Parliament
The case of the small Margaux was told by the same parents in Brussels in front of the plenary room of the European Parliament. The audience gathered for the conference "European research and innovation in our daily lives", Organized by Eurocopter in collaboration with the community executive, he was touched by the story of the girl.
Professor Luigi Naldini, said at the conference that the case of Margaux "it's the story of a dream of a new drug coming true. His genes have been captured, corrected and reinserted. And now, they produce blood cells that work. His case is one of the first successful treatments. After his gene therapy was put to the test in the world, with more than 100 patients only in Milan". The researcher also stressed the importance of research and fundraising.
These latter aspects have also been underlined by the President of the European Parliament, Antonio Tajani who in his speech said: "Without innovation and research, we are not growing, we are not competitive, our citizens are not guaranteed at best."And proposes to increase the funds allocated to research.
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