Article

Long-Term Safety and Effectiveness of Velaglucerase Alfa in Gaucher Disease: 6-Year Interim Analysis of A Post-Marketing Surveillance in Japan

2021-06-10

Abstract excerpt

<title>Abstract</title> <p>Background Gaucher disease (GD) is caused by reduced lysosomal enzyme β-glucocerebrosidase activity. Heterogeneous genotypes and phenotypes have been observed within GD types and across ethnicities. Enzyme replacement therapy is generally recommended for patients with Type 1 GD, the least severe form of GD. In Japan, velaglucerase alfa has a broad indication covering Type 1, 2 or 3 GD....

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: a0a893ed-da8c-5d40-a668-0a81bdaf8975
DOI: 10.21203/rs.3.rs-563813/v1

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

Long-Term Safety and Effectiveness of Velaglucerase Alfa in Gaucher Disease: 6-Year Interim Analysis of A Post-Marketing Surveillance in JapanDOI 10.21203/rs.3.rs-563813/v1Read

Demographics and patient characteristics of 1209 patients with Gaucher disease: Descriptive analysis from the Gaucher Outcome Survey (GOS).Semantic proximity Read

Long-term hematological, visceral, and growth outcomes in children with Gaucher disease type 3 treated with imiglucerase in the International Collaborative Gaucher Group Gaucher Registry.Semantic proximity Read

The SPARKLE registry: protocol for an international prospective cohort study in patients with alpha-mannosidosisSemantic proximity Read

An Overview of Gaucher DiseaseSemantic proximity Read

Recent advances in the diagnosis and management of Gaucher disease.Semantic proximity Read

[Therapeutic objectives in Gaucher disease].Semantic proximity Read

Diagnosis and Management of Gaucher Disease in India – Consensus Guidelines of the Gaucher Disease Task Force of the Society for Indian Academy of Medical Genetics and the Indian Academy of PediatricsSemantic proximity Read

Gaucher disease: recommendations on diagnosis, evaluation, and monitoring.Semantic proximity Read

Changing clinical manifestations of Gaucher disease in TaiwanSemantic proximity Read

Therapeutic goals in Gaucher disease.Semantic proximity Read

The Gaucher registry: demographics and disease characteristics of 1698 patients with Gaucher disease.Semantic proximity Read

Management and monitoring recommendations for the use of eliglustat in adults with type 1 Gaucher disease in Europe.Semantic proximity Read