Facing repeated funding denials from Ontario, a Brampton family is raising millions of dollars for an American gene therapy to treat their two-year-old child’s rare neurological condition.
Zoravar Singh’s AADC Deficiency Diagnosis
Two-year-old Zoravar Singh lives with aromatic L-amino acid decarboxylase deficiency, known as AADC Deficiency, which impairs the brain’s ability to produce an enzyme necessary for neurotransmitters. The condition severely impacts motor control, communication, and behavior, trapping the toddler in a cycle of daily medications and intense physical crises. “His day starts with the medication and ends with the medications,” Ramandeep Kaur told CityNews Toronto.
The physical toll on the child involves severe, prolonged episodes that disrupt everyday life. “Zoravar goes through odysseys, his eyes roll up all the way to the sky, and it stays almost 10 to 15 minutes. Sometimes it stays longer, like about an hour or half an hour,” Kaur explained to CityNews Toronto.
“He used to cry the whole day and whole night. It was so hard for us to handle him when we [would] give him the medication, to move his body.”
FDA Approved Kebilidi Delivers Functional Genetic Material to Brain Cells
The family’s only remaining hope lies in a specialized gene therapy available in Boston, Massachusetts. In November 2024, the U.S. Food and Drug Administration approved the gene therapy Kebilidi for this rare disorder. The intervention involves a single surgical administration where a viral vector delivers functional genetic material directly into targeted brain cells to restore enzyme production.
“One time treatment only for the eight hours a day, and they will insert the medications with the machines into his brain, the cells that are not working, and they put the virus in the brain, and they will start working after that,” Kaur told CityNews Toronto. Despite these technical possibilities, Ontario’s Ministry of Health has repeatedly denied the family’s out-of-country funding applications.
In an official statement, Ontario’s Minister of Health said that Health Canada is solely responsible for determining whether a drug or therapy is authorized for use in Canada, including assessments of safety, efficacy, and quality.
The ministry added that Kebilidi has not been submitted to Health Canada for market approval. Provincial out-of-country treatment policies exclusively fund therapies that would otherwise be covered locally but face procedural delays, meaning unapproved therapies fail to qualify.
Gene Transfer Therapy Introduces Working Genes to Produce Neurotransmitters
- Enzyme Replacement via Gene Transfer: The therapy introduces a working gene using a modified, harmless virus to help the brain produce missing neurotransmitters like dopamine and serotonin.
- Regulatory Status: Because the treatment lacks Health Canada market authorization and provincial funding approval, families must seek alternative financing routes.
Community Fundraising Efforts in Brampton
To bridge the financial gap, the family launched a GoFundMe campaign with a target of $5.5 million to cover the total cost of the American medical intervention. As part of a two-year effort to secure a better quality of life for their son, the community response has already driven fundraising totals past $1.6 million.
The family of two-year-old Zoravar Singh is asking for help for treatment of his rare condition after being denied out-of-country coverage. CityNews Toronto reported the ongoing financial campaign as the family races to secure the remaining funds required for the procedure in Boston.
Contraindications & When to Consult a Doctor
| Parameter | Clinical Detail |
|---|---|
| Condition | AADC Deficiency (Aromatic L-amino acid decarboxylase deficiency) |
| Target Therapy | Kebilidi (eladocagene exuparvovec) |
| Regulatory Status (US) | FDA approved in November 2024 |
| Regulatory Status (Canada) | Not submitted to Health Canada for market approval |
| Fundraising Goal | $5.5 million |
References
- CityNews Toronto reports on Zoravar Singh.
