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Systemic Treatment of Fabry Disease Using a Novel AAV9 Vector Expressing α-Galactosidase A.
Mol Ther Methods Clin Dev. 2020 Oct 22;20:1-17. doi: 10.1016/j.omtm.2020.10.016. eCollection 2021 Mar 12.
Mol Ther Methods Clin Dev. 2020.
PMID: 33335943
Free PMC article.
Fabry disease in the Spanish population: observational study with detection of 77 patients.
Vieitez I, Souto-Rodriguez O, Fernandez-Mosquera L, San Millan B, Teijeira S, Fernandez-Martin J, Martinez-Sanchez F, Aldamiz-Echevarria LJ, Lopez-Rodriguez M, Navarro C, Ortolano S.
Vieitez I, et al. Among authors: souto rodriguez o.
Orphanet J Rare Dis. 2018 Apr 10;13(1):52. doi: 10.1186/s13023-018-0792-8.
Orphanet J Rare Dis. 2018.
PMID: 29631605
Free PMC article.
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