The family of a seriously ill two-month-old baby states that she can now help her, after meeting, to donate more than $ 2 million to help her to become the "most expensive drug in the world". Buy.
Matilde, two months old, was diagnosed with type 1 spinal muscular atrophy six weeks after birth, a genetic disease affecting one in every 11,000 births, which weakens muscles and causes severe mobility problems.
Babies with type 1 ADS have very weak and flaccid arms and legs and have difficulty moving, eating and swallowing. Children born with neurodegenerative disease usually die in the first years of life, usually with severe breathing problems.
But a new drug gives hope to Matilde. May 24, United States The Food and Drug Administration has launched a new Novartis gene therapy called Zolgensma. The only problem – it has a price of 2.1 million.
In order not to be put off, Matilde's parents, who live in Portugal, have launched a fundraising campaign to treat their daughter. They created a Facebook page called "Matilde, a special baby", explaining the difficult situation of their daughter and publicly announcing her bank details.
The site became viral throughout the country and received more than 200,000 "likes" and "followers" as people banded together to raise money for Matilde. The campaign was supported by a number of prominent personalities in Portugal, including television presenter Manuel Luís Goucha and professional cyclist Rui Costa.
The parents said Tuesday that they had not only achieved their heir goal, but that they had even surpassed after raising $ 2.3 million in just two months . Matilde's parents said that they had posted on the Facebook page: "There are still no words in the dictionary that define everything they've done for us.
"We thank you again and we will continue to thank you for your affection and our country is too small for the size of our hearts," they added.
The family said that any money remaining after paying Matilde's treatment would be given to "other families" with the same problem.
Matilde is currently hospitalized at Santa Maria de Lisboa Hospital in Lisbon, the country's capital, and may not be good enough to travel to America for treatment, according to the ABC. However, the family has no choice, the drug has not yet been approved by the European Medicines Agency.
AveXis, a subsidiary of Novartis that manufactures Zolgensma, has contacted Matilde's parents to ask the European Medicines Agency to make an exception so that it can be processed in their home country, reports a resident of Portugal.
Zolgensma has already been used to save the lives of 19 babies in the United States since it was made public, said a resident of Portugal.