The diagnostic journey for patients with late-onset GM2 Gangliosidoses.

Publication Year: 2023

DOI:
10.1016/j.ymgmr.2023.101014

PMCID:
PMC10694732

PMID:
38053937

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
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Core Indicators
Data Sharing
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Evidence found in paper:

"Declaration of Competing Interest MCL, RZ, and IB are employees of Sanofi and hold Sanofi stock or stock options. JB and RG are employees of Fulcrum Research Group, which conducted this research with funding from Sanofi. CT reports research funding from Sanofi to her institution for the AMETHIST trial."

Evidence found in paper:

"Funding Sanofi, Cambridge, MA, USA funded this study. Fulcrum, Waltham, MA, USA designed and conducted the study and analyzed the data. All authors agreed to submit the article for publication."

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

Tool: rtransparent

OST Version: N/A

Last Updated: Aug 05, 2025