Omaveloxolone for the treatment of Friedreich ataxia: clinical trial results and practical considerations

Author:

Lynch David R1,Perlman Susan2,Schadt Kim3

Affiliation:

1. Friedrech Ataxia Program, Division of Neurology, The Children’s Hospital of Philadelphia, Philadelphia, PA, USA

2. Department of Neurology, David Geffen UCLA School of Medicine, Los Angeles, CA, USA

3. Friedreich Ataxia Program, Division of Neurology, The Children’s Hospital of Philadelphia, Philadelphia, PA, USA

Funder

No funding

Publisher

Informa UK Limited

Subject

Pharmacology (medical),Neurology (clinical),General Neuroscience

Reference76 articles.

1. Bidichandani SI, Delatycki MB. Friedreich Ataxia. In: Adam M, Mirzaa G, Pagon R, Wallace S, Bean L, Gripp K Amemiya A, editors. GeneReviews® [internet]. Seattle (WA): University of Washington, Seattle; 1998 Dec 18 [updated 2017 Jun 1]. p. 1993–2023.

2. Friedreich's Ataxia: Autosomal Recessive Disease Caused by an Intronic GAA Triplet Repeat Expansion

3. Friedreich Ataxia: Multidisciplinary Clinical Care

4. Epigenetic promoter silencing in Friedreich ataxia is dependent on repeat length

5. DNA sequence-specific polyamides alleviate transcription inhibition associated with long GAA{middle dot}TTC repeats in Friedreich's ataxia

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