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5-year-old's terminal MLD diagnosis offers brother hope
Two children with metachromatic leukodystrophy, a rare genetic disorder, have been diagnosed in a Long Beach family. The older girl, Anna, shows severe symptoms and is unlikely to benefit from a newly approved gene therapy due to advanced decline, while her younger brother Joey is in early stages and may be saved with the treatment. The therapy, costing $4.25 million, was approved in the US in 2024 and has shown promising results in clinical trials, but its long-term effectiveness remains uncertain. Doctors and parents are racing against time to start Joey’s treatment, as early intervention offers the best chance to prevent severe disability or death.
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