Fundraising Push for Child with Arginase Deficiency

Three-year-old Mylah Algaradi battles Arginase deficiency as her family seeks costly treatment not provided by the NHS.

Fundraising Challenge for Child with Arginase Deficiency

Three-year-old Mylah Algaradi was diagnosed at birth with Arginase deficiency, a rare metabolic disorder that is part of the urea cycle. After a period of stability, her mobility and speech began to deteriorate rapidly, leaving her unable to walk or talk and prompting a transplant plan at Leeds Children's Hospital.

Pegzilarginase, a treatment that could slow or reverse progression, costs £4,600 per vial, about £56,000 for a 12-week course, and is not available on the NHS. The family has applied for compassionate access with the drug maker and with Alder Hey, but both were denied. A GoFundMe page has raised around £18,000, while the family still needs tens of thousands more. Mylah is on a waiting list for a liver transplant at Leeds Children's Hospital since January 2024, with a potential donor uncle.

Rising drug costs test equity in the health system. This case shows how families must navigate a patchwork of funding, charity, and clinical need when rare diseases strike early. Crowdfunding is filling gaps, but it is not a substitute for systematic access to essential medicines.

Policy makers face a hard choice: protect public budgets while ensuring that rare patients do not lose their chance at treatment. The story also highlights how families bear not just medical stress but financial risk, with urgent need of timely support that scales as the disease advances.

Highlights

The story prompts reflection on how health systems decide which medicines reach those in need.