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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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Patient-Reported Symptomatology in Hypophosphatasia
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Polymorphic variants of alkaline phosphatase gene correlate with clinical signs of adult hypophosphatasia?
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Hypophosphatasia and Your Kidneys—What to Expect
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Study supports gene therapy as a promising treatment for soft bone disease
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Gene therapy using adeno-associated virus serotype 8 encoding TNAP-D 10 improves the skeletal and dentoalveolar phenotypes in Alpl -/- mice
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Perinatal hypophosphatasia presenting as neonatal epileptic encephalopathy with abnormal neurotransmitter metabolism secondary to reduced cofactor pyridoxal 5 phosphate availability
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POS1126 PREVALENCE OF HYPOPHOSPHATASIA IN ADULT RHEUMATOLOGY PATIENTS SCREENED BY LOW LEVELS OF ALKALINE PHOSPHATASE
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