This is Maria, the adorable 9-year-old girl with a rare disease and she wants nothing more than to become a doctor to help other people.
But she needs serious surgery in the USA that Pakistan cannot give her.
International Rare Disease Day is on Saturday (29th February) and Maria desperately needs surgery because she suffers from Morquio A syndrome (also called MPS IVA).
It is an autosomal recessive disorder, one of 50 lysosomal storage diseases. It causes the body to be unable to process certain types of sugar molecules. It occurs in 1 in 200,000 to 300,000 newborns. Children with the more severe form of MPS IV may not live beyond their twenties or thirties.
There is no cure for Morquio A, although there is a very expensive drug called Vimizim which costs hundreds of thousands of pounds. Its effectiveness is unclear, and experts say the best thing for Maria is to have the surgery.
Maria’s dad Shahid Ullah speaks English and has been teaching his adorable daughter how to speak it, as can be seen in video 1.
He told Central European News (CEN): “Maria has a progressive disease, she already did cervical spine fusion surgery and she needs also a surgery on both her hips and knees.
“There is no cure for Morquio A, but this disease does have an approved drug which give patients weekly enzyme replacement therapy, Maria is not taking Vimizim.
“Unfortunately we can’t afford this treatment and the government does not support to get Vimizim here in Pakistan.”
When asked about where he would take Maria for treatment, Shahid said: “To DuPont Children’s Hospital in Wilmington, in the state of Delaware, in the USA. Dr William Mackenzie is the best doctor at the best hospital for Morquio A patients.”
Maria loves to play with her siblings. She loves to play on the swings and seesaws and is proud to show off her mastery of the English language. Shahid said: “She loves to play with the dolls with her sister and cousins. She like the cartoon on Snow White, she likes English subject, and talking in English with others.”
Maria’s friends and family have already raised 13,500 USD of the 25,000 USD they need on their GoFundMe page (https://www.gofundme.com/f/k42vy6-maria-walks-for-life) but they still need more donations so Maria can have her surgery.
Speaking to CEN about the GoFundMe page, Shahid said: “We really need more support from people. Many people world wide did help us already. We are extremely grateful for that. It shows that there are so many good willing people on our planet.”
When asked about how Maria copes with the disease, Shahid said: “Maria is now 9 years old, she has some problems due to her illness.
“She has two kinds of physical issues as firstly she can’t walk for long distances and can’t do some things compared to children of her age, and secondly it is hard for her to deal with her illness and accept things.
“It is not easy, but she is humble, kind and thinking about everyone. She is a young girl and depends on her parents, but at the same time she is brave and strong and it’s her who gives us power to fight for her.”
When asked what Maria wants to do when she grows up, Shahid told CEN: “She says she want to become a doctor and help those who need help.”
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