EPISODE · Mar 27, 2026 · 7 MIN
Two decade wait for medicines for rare genetic disorder
from Checkpoint
A group representing people with a rare genetic disorder say they've been waiting almost two decades for some critical drugs to be funded. Fabry disease is caused by a missing enzyme, and can lead to life threatening complications including organ failure. There is no cure but there are treatments available overseas to slow degeneration from the disease. Deborah Klyer from Fabry New Zealand spoke to Lisa Owen.
What this episode covers
A group representing people with a rare genetic disorder say they've been waiting almost two decades for some critical drugs to be funded. Fabry disease is caused by a missing enzyme, and can lead to life threatening complications including organ failure. There is no cure but there are treatments available overseas to slow degeneration from the disease. Deborah Klyer from Fabry New Zealand spoke to Lisa Owen.
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Two decade wait for medicines for rare genetic disorder
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