Vestronidase - Latest Advances

Enzyme Replacement Therapy in Mucopolysaccharidosis Type VII: A Three-Year Clinical Outcome Study of the First Taiwanese Case.

Journal: Diagnostics (Basel, Switzerland)

Published: December 16, 2024

Potential Targeting Mechanisms for Bone-Directed Therapies.

Journal: International journal of molecular sciences

Published: May 21, 2024

Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program.

Journal: Orphanet journal of rare diseases

Published: December 17, 2023

Prenatal diagnosis of mucopolysaccharidosis type VII facilitating treatment in neonatal period.

Journal: Prenatal diagnosis

Published: September 06, 2023

Mucopolysaccharidosis type VII (Sly syndrome) - What do we know?

Journal: Molecular genetics and metabolism

Published: August 22, 2023

Exploring the feasibility of using the ICER Evidence Rating Matrix for Comparative Clinical Effectiveness in assessing treatment benefit and certainty in the clinical evidence on orphan therapies for paediatric indications.

Journal: Orphanet journal of rare diseases

Published: January 04, 2023

Dose-dependent effects of enzyme replacement therapy on skeletal disease progression in mucopolysaccharidosis VII dogs.

Journal: Molecular therapy. Methods & clinical development

Published: August 22, 2022

Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII.

Journal: Molecular genetics and metabolism

Published: November 05, 2021

Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII.

Journal: Molecular genetics and metabolism reports

Published: February 04, 2021

The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases.

Journal: Orphanet journal of rare diseases

Published: September 08, 2020

Corrigendum to "The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII" [Mol Genet Metab 2020 Mar;129(3):219-227].

Journal: Molecular genetics and metabolism

Published: August 27, 2020

Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations.

Journal: International journal of molecular sciences

Published: March 19, 2020

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Enzyme Replacement Therapy in Mucopolysaccharidosis Type VII: A Three-Year Clinical Outcome Study of the First Taiwanese Case.

Enzyme Replacement Therapy in Mucopolysaccharidosis Type VII: A Three-Year Clinical Outcome Study of the First Taiwanese Case.

Potential Targeting Mechanisms for Bone-Directed Therapies.

Potential Targeting Mechanisms for Bone-Directed Therapies.

Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program.

Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program.

Prenatal diagnosis of mucopolysaccharidosis type VII facilitating treatment in neonatal period.

Prenatal diagnosis of mucopolysaccharidosis type VII facilitating treatment in neonatal period.

Mucopolysaccharidosis type VII (Sly syndrome) - What do we know?

Mucopolysaccharidosis type VII (Sly syndrome) - What do we know?

Exploring the feasibility of using the ICER Evidence Rating Matrix for Comparative Clinical Effectiveness in assessing treatment benefit and certainty in the clinical evidence on orphan therapies for paediatric indications.

Exploring the feasibility of using the ICER Evidence Rating Matrix for Comparative Clinical Effectiveness in assessing treatment benefit and certainty in the clinical evidence on orphan therapies for paediatric indications.

Dose-dependent effects of enzyme replacement therapy on skeletal disease progression in mucopolysaccharidosis VII dogs.

Dose-dependent effects of enzyme replacement therapy on skeletal disease progression in mucopolysaccharidosis VII dogs.

Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII.

Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII.

Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII.

Open-label phase 1/2 study of vestronidase alfa for mucopolysaccharidosis VII.

The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases.

The multi-domain responder index: a novel analysis tool to capture a broader assessment of clinical benefit in heterogeneous complex rare diseases.

Corrigendum to "The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII" [Mol Genet Metab 2020 Mar;129(3):219-227].

Corrigendum to "The long-term safety and efficacy of vestronidase alfa, rhGUS enzyme replacement therapy, in subjects with mucopolysaccharidosis VII" [Mol Genet Metab 2020 Mar;129(3):219-227].

Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations.

Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations.