Baby Pia, suffering from a rare genetic disease, received her medicine, said the mother of the child at a press conference on Friday. The mother reassured the general public and the many donors that everything had gone well.
10-month-old Pia, who is suffering from spinal muscular atrophy, a rare genetic disease, received Zolgensma treatment on Wednesday at the University Hospital of Antwerp (UZA), the mother of the child, Ellen De Meyer, at a press conference. Everything went well, she said.
Spinal muscular atrophy is fatal for young children because it causes muscle atrophy, which in particular prevents the lungs from functioning. Baby Pia received a bite of Zolgensma, a curative medicine. A major fundraiser was launched in September to raise 1.9 million euros, the cost of a single injection of Zolgensma. This treatment, developed by the Swiss pharmaceutical firm Novartis, had not yet been approved by the European Medicines Agency (EMA). It's done since Wednesday. "Everything went faster than expected," commented Ellen De Meyer. "We arrived at the hospital Monday night and the sting was administered Wednesday. Pia was put on an infusion for one hour. We hope to be able to go home this weekendPia shows no sign of a side effect, 48 hours after the injection, and the whole family is still waiting for the first results. "She should first manage to hold her head straight; then we hope she will have more strength in the bust "said the mother of the child. In Pia's case, the treatment will not cure her completely but should greatly increase her quality of life by allowing her to sit autonomously, the parents hope. "She will then be able to move around in a wheelchair and fully live her life"they concluded.
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