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. 2024 Feb 3;30(2):201-205.
doi: 10.18553/jmcp.2024.30.2.201.

Atidarsagene autotemcel for metachromatic leukodystrophy

Shahariar Mohammed Fahim  1 , Grace Lin  1   2 , Kangho Suh  3 , Josh J Carlson  4 , Marina Richardson  1 , Belen Herce-Hagiwara  1 , Ronald Dickerson  4 , Steven D Pearson  1 , David M Rind  1 , Foluso Agboola  1
Affiliations

Atidarsagene autotemcel for metachromatic leukodystrophy

Shahariar Mohammed Fahim et al. J Manag Care Spec Pharm. .
No abstract available

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References

    1. Gomez-Ospina N. Arylsulfatase A deficiency. In: Adam MP, Everman DB, Mirzaa GM, et al., eds. GeneReviews(®). University of Washington, Seattle. 1993-2023:1-26.
    1. MLD Foundation. Analysis of MLD Incidence & Prevalence: Domestic & Worldwide. Published 2017. Updated February 2022. Accessed April 4, 2023. https://MLD.foundation/Incidence
    1. Elgün S, Waibel J, Kehrer C, et al. . Phenotypic variation between siblings with metachromatic leukodystrophy. Orphanet J Rare Dis. 2019;14(1):136. doi:10.1186/s13023-019-1113-6 - PMC - PubMed
    1. Shaimardanova AA, Chulpanova DS, Solovyeva VV, et al. . Metachromatic leukodystrophy: Diagnosis, modeling, and treatment approaches. Front Med (Lausanne). 2020;7:576221. doi:10.3389/fmed.2020.576221 - PMC - PubMed
    1. van Rappard DF, Boelens JJ, Wolf NI. Metachromatic leukodystrophy: Disease spectrum and approaches for treatment. Best Pract Res Clin Endocrinol Metab. 2015;29(2):261-73. doi:10.1016/j.beem.2014.10.001 - PubMed