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Author

Jaco Botha

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2 papers indexed here

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Open access Oct 2026

Safety, Efficacy, and Pharmacokinetics of Agalsidase Alfa in Treatment-Naive Chinese Patients with Fabry Disease: 52-Week Phase 3, Open-Label, Single-Arm Study.

INTRODUCTION Clinical data on the efficacy, tolerability, and pharmacokinetics of agalsidase alfa in Chinese patients with Fabry disease are limited. We evaluated the safety, efficacy, and pharmacokinetics of agalsidase alfa in Chinese patients who are treatment-naive over 52 weeks. METHODS This was an open-label, no...

Hong Ren, Jian-Hua Mao, Rong Wang et al. · 0 citations
Review Oct 2026

Clinical characteristics and management of patients with mucopolysaccharidosis II in the United States enrolled in the Hunter Outcome Survey.

BACKGROUND In 2022, mucopolysaccharidosis II (MPS II) was added to the list of disorders recommended for inclusion in newborn screening panels in the USA; this is expected to substantially facilitate earlier diagnosis and treatment and improve patient outcomes. An analysis was conducted in US patients enrolled in the H...

Barbara K. Burton, C. Ficicioglu, Helio F. Pedro et al. · 0 citations

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