Survival of patients with chronic acid sphingomyelinase deficiency (ASMD) in the United States: A retrospective chart review study.

Authors:
Pulikottil-Jacob R; Dehipawala S; Smith B; Athavale A; Gusto G and 7 more

Journal:
Mol Genet Metab Rep

Publication Year: 2023

DOI:
10.1016/j.ymgmr.2023.101040

PMCID:
PMC10767269

PMID:
38188692

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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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"Declaration of Competing Interest MVMR, MF, RPJ, SG, and LP are employees and shareholders of Sanofi. SD, BS, and AA are employees of Trinity Life Sciences. GG, AC, AK, and TB are employees of Certara, which received funding from Sanofi for this research."

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"Funding This study was funded by 10.13039/100004339Sanofi."

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Last Updated: Aug 05, 2025