Results From a 12-Month Open-Label Phase 1/2 Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease-Reference-Cited by-同舟云学术

Results From a 12-Month Open-Label Phase 1/2 Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

Published:2018-01 Issue: Volume:6 Page:232640981876556
ISSN:2326-4098
Container-title:Journal of Inborn Errors of Metabolism and Screening
language:en
Short-container-title:Journal of Inborn Errors of Metabolism and Screening

Author:

Tantawy Azza A. G.¹,El-Beshlawy Amal²,Marzouk Iman³,Bavdekar Ashish⁴,Qin Yulin⁵,Mellgard Björn⁶,Ben Turkia Hadhami⁷

Affiliation:

1. Pediatric Department, Faculty Of Medicine, Ain Shams University Hospital, Heliopolis, Cairo, Egypt

2. Department of Pediatric Hematology, Cairo University Hospital, Cairo, Egypt

3. Faculty of Medicine, Alexandria University Hospital, Alexandria, Egypt

4. KEM Hospital Research Centre, Rasta Peth, Pune, Maharashtra, India

5. Shire, Lexington, MA, USA

6. Shire, Zug, Switzerland

7. La Rabta Hospital, Tunis, Tunisia

Funder

Shire Human Genetic Therapies, Inc.

Publisher

FapUNIFESP (SciELO)

Subject

Genetics(clinical),Endocrinology, Diabetes and Metabolism,Pediatrics, Perinatology, and Child Health

Link

http://journals.sagepub.com/doi/pdf/10.1177/2326409818765564

Reference26 articles.

1. Beutler E, Grabowski G. Gaucher disease. In: Scriver CR, Beaudet AL, Sly WS, Valle D, ed. The Metabolic and Molecular Bases of Inherited Diseases. 8th ed. New York: McGraw-Hill; 2001:3635–3668.

2. The Gaucher Registry

3. Neuronopathic Gaucher disease: demographic and clinical features of 131 patients enrolled in the International Collaborative Gaucher Group Neurological Outcomes Subregistry

4. The Spectrum of Neurological Manifestations Associated with Gaucher Disease

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