Combining angiotensin receptor blockade and enzyme replacement therapy for vascular disease in mucopolysaccharidosis type I.

Authors:
Hurt SC; Vera MU; Le SQ; Kan SH; Bui Q and 1 more

Journal:
Mol Genet Metab Rep

Publication Year: 2023

DOI:
10.1016/j.ymgmr.2023.101036

PMCID:
PMC10761904

PMID:
38173710

Journal Information

Full Title: Mol Genet Metab Rep

Abbreviation: Mol Genet Metab Rep

Country: Unknown

Publisher: Unknown

Language: N/A

Publication Details

Subject Category: Genetics & Heredity

Available in Europe PMC: Yes

Available in PMC: Yes

PDF Available: No

Transparency Score
3/6
50.0% Transparent
Transparency Indicators
Click on green indicators to view evidence text
Core Indicators
Data Sharing
Code Sharing
Evidence found in paper:

"Declaration of Competing Interest This study was supported by a grant from Sanofi Corporation. Dr. Dickson is a listed inventor on Patent #USSN 15/946,505; 0WVR-223,143-US for enzyme replacement therapy for mucopolysaccharidosis IIID. Dr. Dickson also receives research support from M6P Therapeutics, Alnylam, and Mandos Health."

Evidence found in paper:

"Funding for this study was provided by Sanofi Corporation, the 10.13039/100013927National MPS Society, and the Lysosomal Disease Network. The Lysosomal Disease Network (LDN) U54-NS065768 is a part of the National Institute of Health (NIH) Rare Diseases Clinical Research Network, supported through collaboration between the NIH Office of Rare Diseases Research at the National Center for Advancing Translational Science, the National Institute of Neurological Disorders and Stroke (NINDS), and National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK). The content is solely the responsibility of the authors and does not necessarily represent the official views of the NIH. Funding was also provided by the National Science Foundation (NSF) Graduate Research Fellowship Program DGE-1745038 and National Heart, Lung, and Blood Institute (NHLBI) F31 HL165903-01A1 to S. H. S.-h.K. was supported by a T32 fellowship in the UCLA Medical Genetics Training Program (GM008243). rhIDU in the form of laronidase was donated by Sanofi Corporation. Sarah C. Hurt: Formal analysis, Investigation, Writing – original draft, Writing – review & editing. Moin U. Vera: Conceptualization, Funding acquisition, Methodology, Writing – review & editing. Steven Q. Le: Investigation, Methodology, Writing – review & editing. Shih-hsin Kan: Investigation, Methodology, Writing – review & editing. Quang Bui: Investigation, Methodology. Patricia I. Dickson: Conceptualization, Formal analysis, Funding acquisition, Methodology, Project administration, Resources, Supervision, Writing – review & editing. This study was supported by a grant from Sanofi Corporation. Dr. Dickson is a listed inventor on Patent #USSN 15/946,505; 0WVR-223,143-US for enzyme replacement therapy for mucopolysaccharidosis IIID. Dr. Dickson also receives research support from M6P Therapeutics, Alnylam, and Mandos Health."

Protocol Registration
Open Access
Paper is freely available to read
Additional Indicators
Replication
Novelty Statement
Assessment Info

Tool: rtransparent

OST Version: N/A

Last Updated: Aug 05, 2025