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Alexion Reports Positive Phase 3 Results for Efzimfotase Alfa in Children with Rare Bone Disease
Alexion, AstraZeneca Rare Disease, has announced positive Phase 3 results for investigational enzyme replacement therapy efzimfotase alfa in children with HPP. The therapy demonstrated significant improvements in bone health and physical function, reinforcing its potential as a next-generation treatment for the rare inherited metabolic disorder.
Rickets Severity Score | 30/06/2026 | By News Bureau
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