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Contemporary therapeutics and new drug developments for treatment of Fabry disease: a narrative review

Fabry disease (OMIM 301500) is an X-linked (Xq22.1) lysosomal storage disorder leading to a progressive multisystem disease with high variability in both genotype and phenotype expression. The pathophysiological origin is found in an enzyme deficiency of the α-galactosidase A (enzyme commission no....

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Vydáno v:Cardiovasc Diagn Ther
Hlavní autoři: Oder, Daniel, Müntze, Jonas, Nordbeck, Peter
Médium: Artigo
Jazyk:Inglês
Vydáno: AME Publishing Company 2021
Témata:
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC8102271/
https://ncbi.nlm.nih.gov/pubmed/33968645
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.21037/cdt-20-743
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