Jaco A. Botha
Glattpark, ZH, Switzerland
Last Updated: 09/26/2026
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Found 49 publications
A 53-week, open-label phase IIIb study of velaglucerase alfa in Chinese patients with type 1 Gaucher disease: Safety, efficacy, and pharmacokinetics.
Journal: Molecular genetics and metabolism reports
Published: March 02, 2026
Improvement of Bone Mineral Density in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: Results from Clinical Studies.
Journal: Journal of clinical medicine
Published: January 19, 2026
Clinical characteristics and real-world outcomes in patients with mucopolysaccharidosis II over 18 years: final report of the Hunter Outcome Survey.
Journal: Molecular genetics and metabolism
Published: July 04, 2025
Long-term effectiveness and safety outcomes in adults with Fabry disease treated with agalsidase alfa: 20 years of data from the Fabry Outcome Survey.
Journal: European journal of clinical investigation
Published: June 26, 2025
Safety profile of idursulfase administered at home in patients with mucopolysaccharidosis II enrolled in the Hunter Outcome Survey.
Journal: Molecular genetics and metabolism
Published: June 17, 2025
Female Patients With Mucopolysaccharidosis II (MPS II): Insights From the Hunter Outcome Survey.
Journal: JIMD reports
Published: April 25, 2025
Two decades of experience of the Fabry Outcome Survey provides further confirmation of the long-term effectiveness of agalsidase alfa enzyme replacement therapy.
Journal: Molecular genetics and metabolism reports
Published: January 22, 2025
Unmet needs of adults living with mucopolysaccharidosis II: data from the Hunter Outcome Survey.
Journal: Orphanet journal of rare diseases
Published: June 27, 2024
Genotype-phenotype findings in patients with mucopolysaccharidosis II from the Hunter Outcome Survey.
Journal: Molecular genetics and metabolism
Published: May 22, 2024
Twelve Years of the Gaucher Outcomes Survey (GOS): Insights, Achievements, and Lessons Learned from a Global Patient Registry.
Journal: Journal of clinical medicine
Published: April 19, 2024
Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry.
Journal: Journal of clinical medicine
Published: March 25, 2024
Safety analysis of self-administered enzyme replacement therapy using data from the Fabry Outcome and Gaucher Outcome Surveys.
Journal: Orphanet journal of rare diseases
Published: March 12, 2024
Last Updated: 09/26/2026