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Literature DB >> 26782317

Emerging therapies in Friedreich's ataxia.

Tanya V Aranca¹, Tracy M Jones¹, Jessica D Shaw¹, Joseph S Staffetti¹, Tetsuo Ashizawa², Sheng-Han Kuo³, Brent L Fogel⁴, George R Wilmot⁵, Susan L Perlman⁶, Chiadi U Onyike⁷, Sarah H Ying^8,9,10, Theresa A Zesiewicz^1,11.

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.

Entities: Chemical Disease Gene Mutation Species

Keywords: FRDA; Friedreich's ataxia; HDAC; antioxidants; erythropoietin; iron

Mesh：

Substances：
Nerve Growth Factors

Year: 2016 PMID： 26782317 PMCID： PMC4768799 DOI： 10.2217/nmt.15.73

Source DB: PubMed Journal: Neurodegener Dis Manag ISSN： 1758-2024

78 in total

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Journal: Neurology Date: 2012-10-31 Impact factor: 9.910

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Authors: Lingli Li; Lucille Voullaire; Chiranjeevi Sandi; Mark A Pook; Panos A Ioannou; Martin B Delatycki; Joseph P Sarsero
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26 in total

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3. Phosphodiesterase Inhibitors Revert Axonal Dystrophy in Friedreich's Ataxia Mouse Model.

Authors: Belén Mollá; Diana C Muñoz-Lasso; Pablo Calap; Angel Fernandez-Vilata; María de la Iglesia-Vaya; Federico V Pallardó; Maria Dolores Moltó; Francesc Palau; Pilar Gonzalez-Cabo
Journal: Neurotherapeutics Date: 2019-04 Impact factor: 7.620

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Authors: Mehmet Fatih Yetkİn; Murat GÜltekİn
Journal: Noro Psikiyatr Ars Date: 2020-09-21 Impact factor: 1.339

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Authors: Jiun-I Lai; Daniel Nachun; Lina Petrosyan; Benjamin Throesch; Erica Campau; Fuying Gao; Kristin K Baldwin; Giovanni Coppola; Joel M Gottesfeld; Elisabetta Soragni
Journal: J Biol Chem Date: 2018-12-14 Impact factor: 5.157

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Authors: Alessandra Bolotta; Antonella Pini; Provvidenza M Abruzzo; Alessandro Ghezzo; Alessandra Modesti; Tania Gamberi; Carla Ferreri; Francesca Bugamelli; Filippo Fortuna; Silvia Vertuani; Stefano Manfredini; Cinzia Zucchini; Marina Marini
Journal: Exp Biol Med (Maywood) Date: 2019-12-03