Mylah Algaradi seeks funds for life saving drug

Mylah Algaradi’s family in the UK seeks £56,000 for a life-saving drug not funded by the NHS to slow a rare metabolic disorder.

Mum fights to fund life-saving treatment for 3-year-old daughter

Mylah Algaradi, three, was diagnosed at birth with Arginase deficiency, a rare metabolic disorder, and has been supported by Alder Hey Children’s Hospital and Manchester Children’s Metabolic Team. After years of steady progress on a strict low-protein diet, she began to lose the ability to walk and talk this year, and now relies on careful care while awaiting a liver transplant in Leeds.

This case shows how pricey modern therapies can strain public health budgets when coverage isn’t guaranteed. Pegzilarginase costs £4,600 per vial, about £56,000 for a 12-week course, and it is not funded by the NHS. That creates pressure on families and raises questions about how to balance patient need with system limits.
Crowdfunding can spark quick generosity but cannot replace a sustainable plan for rare diseases. While GoFundMe appeals reveal community support, they also highlight gaps in access and equity that public policy must address.

Highlights

As more families face similar costs, policy makers will be watched for real solutions, not elaborate stops in between.