title: Emerging therapies in Friedreich's ataxia.
abstract: Friedreich's ataxia (FRDA) is an inherited, progressive neurodegenerative disease that typically affects teenagers and young adults. Therapeutic strategies and disease insight have expanded rapidly over recent years, leading to hope for the FRDA population. There is currently no US FDA-approved treatment for FRDA, but advances in research of its pathogenesis have led to clinical trials of potential treatments. This article reviews emerging therapies and discusses future perspectives, including the need for more precise measures for detecting changes in neurologic symptoms as well as a disease-modifying agent.
authors.affiliation: University of South Florida Ataxia Research Center, Department of Neurology, FL, USA.
authors.forename: Tanya V
authors.initials: TV
authors.lastname: Aranca
authors.affiliation: University of South Florida Ataxia Research Center, Department of Neurology, FL, USA.
authors.forename: Tracy M
authors.initials: TM
authors.lastname: Jones
authors.affiliation: University of South Florida Ataxia Research Center, Department of Neurology, FL, USA.
authors.forename: Jessica D
authors.initials: JD
authors.lastname: Shaw
authors.affiliation: University of South Florida Ataxia Research Center, Department of Neurology, FL, USA.
authors.forename: Joseph S
authors.initials: JS
authors.lastname: Staffetti
authors.affiliation: McKnight Brain Institute, University of Florida Department of Neurology, FL, USA.
authors.forename: Tetsuo
authors.initials: T
authors.lastname: Ashizawa
authors.affiliation: Department of Neurology, Columbia University, NY, USA.
authors.forename: Sheng-Han
authors.initials: SH
authors.lastname: Kuo
authors.affiliation: Department of Neurology, Neurogenetics Program, David Geffen School of Medicine, University of California, CA, USA.
authors.forename: Brent L
authors.initials: BL
authors.lastname: Fogel
authors.affiliation: Department of Neurology, Emory University, GA, USA.
authors.forename: George R
authors.initials: GR
authors.lastname: Wilmot
authors.affiliation: Ataxia and Huntington Disease Center of Excellence, University of California, CA, US.
authors.forename: Susan L
authors.initials: SL
authors.lastname: Perlman
authors.affiliation: Department of Psychiatry and Behavioral Sciences, Johns Hopkins University school of Medicine MD, USA.
authors.forename: Chiadi U
authors.initials: CU
authors.lastname: Onyike
authors.affiliation: Department of Neurology, Johns Hopkins University School of Medicine, MD, USA.|Department of Radiology and Radiological Sciences, Johns Hopkins University School of Medicine, MD, USA.|Department of Ophthalmology, Johns Hopkins University School of Medicine, MD, USA.
authors.forename: Sarah H
authors.initials: SH
authors.lastname: Ying
authors.affiliation: University of South Florida Ataxia Research Center, Department of Neurology, FL, USA.|James A. Haley Veterans' Hospital, FL, USA.
authors.forename: Theresa A
authors.initials: TA
authors.lastname: Zesiewicz
mesh_terms: D000293:Adolescent; D002986:Clinical Trials as Topic; D004359:Drug Therapy, Combination / Q000639:trends; D005621:Friedreich Ataxia / Q000235:genetics / Q000534:rehabilitation / Q000628:therapy*; D015316:Genetic Therapy / Q000639:trends; D006801:Humans; D009414:Nerve Growth Factors / Q000627:therapeutic use; D012720:Severity of Illness Index; D016896:Treatment Outcome
chemical_list: D009414:Nerve Growth Factors
keywords: FRDA; Friedreich's ataxia; HDAC; antioxidants; erythropoietin; iron