A sick Belgian toddler whose family successfully crowdfunded millions of euros to afford the world's most expensive medicine must still be screened to determine her compatibility with the drug.
An outpour of support from Belgians to a nation-wide crowdfunding campaign saw the family of 9-month old Pia raise the €1.9 million needed to afford Zolgensma, the only drug capable of treating their child's genetic condition and which has yet to receive market approval in Europe.
Pia suffers from spinal muscular atrophy, an aggressive genetic disease which progressively leads to muscular failure, and which sees most children diagnosed with it die in early infancy.
The family, who initially announced they would travel to the United States for their daughter's treatment, have said that their daughter must first be tested to ensure she can be treated with the drug.
"Pia will be tested next week to determine if she is not immune to the treatment she needs," her mother, Ellen De Meyer, said, according to La Libre. "We hope that the results are positive and that Pia will be able to receive the shot this year," she said, referring to the single-dose needed to treat her daughter.
The tests, which will be carried out after a screening test is shipped from the United States, will determine whether Zolgensma can be used to treat Pia.
"If certain antibodies are present, we can't start the treatment — this would have harmful consequences, with severe allergic reactions," Professor Guy Hans of Antwerp University Hospital told VRT, adding that it was not yet certain whether Pia could receive the drug.
In an interview with VTM News, De Meyer also said that they still needed to receive the money from telecom operators, and that, depending on industry and government regulations, their daughter could receive the treatment in the United States, Belgium or elsewhere in Europe.
The Brussels Times