Whole genome sequencing in adults with clinical hallmarks of hypophosphatasia negative for ALPL variants
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Safety, pharmacokinetics, and pharmacodynamics of efzimfotase alfa, a second-generation enzyme replacement therapy: phase 1, dose-escalation study in adults with hypophosphatasia
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Prevalence of chondrocalcinosis and calcium pyrophosphate deposition disease in a cohort of adult patients with low alkaline phosphatase levels and a positive versus negative genetic ALPL study
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Gene Therapy for Hypophosphatasia: Current Management and Future
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Successful treatment with bone maturation and average life expectancy of hypophosphatasia model mice by adeno-associated virus type 8-mediated neonatal gene therapy via single muscle injection
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