The world’s most expensive medication, costing $2.2 million, is the only chance that 11-week-old Violet Fitzgerald has of survival.
The little girl, who suffers from type 1 spinal muscular atrophy, is currently in the Children’s Hospital of Philadelphia with her parents Brooke and Dan Fitzgerald, awaiting a one-off infusion of the gene therapy drug Zolgensma.
She is scheduled to receive the medication during an hour-long infusion on Tuesday, 15 Feb. The younger a baby is when the drug is administered, the more effective it is.
Mom Brooke explained that, without the medication, her daughter will get weaker and weaker, and may not live beyond the age of 2.
After their health insurance company BritCay refused to cover the cost of the therapy, because the insurance policy specifically precluded gene therapy, the Fitzgeralds turned to the government and the Cayman Islands National Insurance Company (CINICO) for help. CINICO approved the coverage and the family travelled from Grand Cayman to Philadelphia late last week.
Violet was an active and alert baby at first, Brooke said, but after a few weeks, her parents realised something was wrong.
“At four weeks, we noticed her arms were placing a little bit funny and she was not moving as much as she was before. I took her to the paediatrician to have her arms looked at. The paediatrician did a full examination and did tests, including for SMA. She said there was a one in 10,000 chance, but that, if she had it, she had to get treated right away.
“We waited two weeks for the test results. We were in panic mode for those two weeks, hoping and thinking that there is no way she has this. Then we were called in by the doctor who said she had it. We had to figure out quickly what we had to do.”
Initially, it seemed her private insurance company would cover the treatment cost by paying out the child’s full lifetime coverage of $2 million, meaning the family still had to find an additional $200,000. However, when it turned out the company would not pay for gene therapy, the family turned to other avenues, including fundraising and approaching the Needs Assessment Unit and CINICO.
Time was of the essence, however; with each passing day of Violet’s life, the potential effectiveness of the Zolgensma therapy was decreasing.
Even after she receives the drug, it’s likely Violet will need additional assistance throughout her life, and may require the use of a wheelchair and ventilators, and possibly full-time care, Brooke said.
Zolgensma and spinal muscular atrophy
SMA is a degenerative and oftentimes fatal condition, that involves muscle weakness, loss of movement and paralysis. It’s the leading genetic cause of death in infants. Children born with SMA are missing a motor neuron protein in their cells that is essential for muscle movement.
The intravenous, single-dose Zolgensma therapy replaces the missing or faulty gene with a functioning gene.
Violet appears to be the only child in the Cayman Islands who will have been treated by this therapy.
Her mother explained that if Violet had been born before this drug was available, “her chances of dying before the age of 2 would be 90%”.
Brooke said she hopes that her family’s story may encourage standard gene testing for pregnant women and newborn babies in Cayman, so that if future cases of SMA occur, then they can be caught early and treated.
Even with CINICO paying for the therapy costs, the Fitzgeralds are facing a multitude of other expenses as well, including accommodation, while they stay in the US with their daughter. They will remain there for at least three months after the drug has been administered, while weekly blood tests and other follow-up checks are done. After that, there will have to be more trips and visits to the hospital.
Friends, family members and other supporters have already donated $45,000 via a Go Fund Me page.
Brooke said BritCay has agreed to pay 80% of other medical non-gene-therapy costs associated with her daughter’s treatment.
Once the drug is given to Violet on Tuesday, the family and the doctors will have to wait and see how effective it is, as each child reacts differently to it, Brooke said.
“She’s 11 weeks old and weighs 11 pounds, and she cannot support her head at all,” she added.
“We don’t know when we’ll start to see an improvement. It might be over each month, little by little. … We’re hoping she responds well and gets stronger day by day.”
The Go Fund Me page for Violet can be found here.
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