Reduction of lysosome abundance and GAG accumulation after odiparcil treatment in MPS I and MPS VI models.

Authors:
Tuyaa-Boustugue P; Jantzen I; Zhang H; Young SP; Broqua P and 2 more

Journal:
Mol Genet Metab Rep

Publication Year: 2023

DOI:
10.1016/j.ymgmr.2023.101011

PMCID:
PMC10694777

PMID:
38053941

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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

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3/6
50.0% Transparent
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Evidence found in paper:

"Declaration of Competing Interest The authors declare the following financial interests/personal relationships which may be considered as potential competing interests. PTB, IJ and PB are employees of Inventiva and receive financial compensations from Inventiva; EE and MT were employees of Inventiva while the study was conducted. HZ and SPY (Duke University) provided services on behalf of Inventiva. Odiparcil has been under development as a product for treatment of Mucopolysaccharidosis by Inventiva."

Evidence found in paper:

"Funding Funding for this research was provided by Inventiva SA."

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Paper is freely available to read
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Last Updated: Aug 05, 2025