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Therapeutic Prospects for Friedreich's Ataxia.
Zhang, Siyuan; Napierala, Marek; Napierala, Jill S.
Affiliation
  • Zhang S; Department of Biochemistry and Molecular Genetics, University of Alabama at Birmingham (UAB), 1825 University Boulevard, Birmingham, AL 35294, USA.
  • Napierala M; Department of Biochemistry and Molecular Genetics, University of Alabama at Birmingham (UAB), 1825 University Boulevard, Birmingham, AL 35294, USA.
  • Napierala JS; Department of Biochemistry and Molecular Genetics, University of Alabama at Birmingham (UAB), 1825 University Boulevard, Birmingham, AL 35294, USA. Electronic address: jsbutler@uab.edu.
Trends Pharmacol Sci ; 40(4): 229-233, 2019 04.
Article in En | MEDLINE | ID: mdl-30905359
Friedreich's ataxia (FRDA) is a progressive disease affecting multiple organs that is caused by systemic insufficiency of the mitochondrial protein frataxin. Current therapeutic strategies aim to elevate frataxin levels and/or alleviate the consequences of frataxin deficiency. Recent significant advances in the FRDA therapeutic pipeline are bringing patients closer to a cure.
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Full text: 1 Database: MEDLINE Main subject: Friedreich Ataxia / Iron-Binding Proteins / Mitochondria Limits: Animals / Humans Language: En Journal: Trends Pharmacol Sci Year: 2019 Type: Article Affiliation country: United States
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Full text: 1 Database: MEDLINE Main subject: Friedreich Ataxia / Iron-Binding Proteins / Mitochondria Limits: Animals / Humans Language: En Journal: Trends Pharmacol Sci Year: 2019 Type: Article Affiliation country: United States