Saturday, November 13, 2021

Friedreich's ataxia. The investigator is awaiting Institutional Review Board approval of the planned Phase 2a clinical trial in patients with Friedreich's ataxia and expects to begin enrollment in early 2022.

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BOSTON, Nov. 11, 2021 /PRNewswire/ -- Stealth Biotherapeutics (Nasdaq:MITO), a clinical-stage biotechnology company focused on the discovery...
Wednesday, November 10, 2021

Clinical Initiation of Lead GeneTACTM Program for Friedreich Ataxia On-track for the First Half of 2022

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CARLSBAD, Calif., Nov. 09, 2021 (GLOBE NEWSWIRE); Design Therapeutics Reports Pipeline Progress and Third Quarter 2021 Results  Friedreich A...
Tuesday, November 9, 2021

Molecular approaches for the treatment and prevention of Friedreich's ataxia

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Wenyao Yang, Bruce Thompson, Faith A.A. Kwa, Drug Discovery Today, 2021, doi:10.1016/j.drudis.2021.11.003 Current management strategies aim ...
Sunday, November 7, 2021

Designing phase II clinical trials in Friedreich ataxia

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Rodden LN, Lynch DR.; Expert Opin Emerg Drugs. 2021 Oct 25. doi: 10.1080/14728214.2021.1998452. Epub ahead of print. PMID: 34693848.  A grow...
Friday, November 5, 2021

Construction of DNA/RNA Triplex Helices Based on GAA/TTC Trinucleotide Repeats

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Zhang J, Fakharzadeh A, Pan F, Roland C, Sagui C.; Bio Protoc. 2021 Sep 20;11(18):e4155. doi: 10.21769/BioProtoc.4155. PMID: 34692905; PMCID...
Thursday, November 4, 2021

Longitudinal investigation of brain activation during motor tasks in Friedreich ataxia: 24-month data from IMAGE-FRDA

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Shishegar R, Harding IH, Selvadurai LP, Corben LA, Delatycki MB, Egan GF, Georgiou-Karistianis N.; Brain Struct Funct. 2021 Oct 23. doi: 10....
Tuesday, November 2, 2021

Clinical and Molecular Features of First Mexican Friedreich's Ataxia Patients with Compound Heterozygous FXN Mutations

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Boll MC, Gasca-Saldaña D, Mayén-Lobo YG, Dávila-Ortiz de Montellano DJ, Monroy-Jaramillo N.; Neurol India 2021;69:1363-7. DOI: 10.4103/0028-...
Saturday, October 30, 2021

Left atrial appendage thrombosis in a patient with Friedreich Ataxia–related cardiomyopathy, left ventricular systolic dysfunction, and atrial fibrillation

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. Michele Russo, Annachiara Nuzzo, Matteo Foschi, Simona Boarin, Stefano Lorenzetti, Corrado Tomasi, Pietro Querzani, Andrea Rubboli. SAGE O...

Tract-Specific Spinal Cord Diffusion Tensor Imaging in Friedreich's Ataxia

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Ana Luisa C.C. Hernandez MD,Thiago J.R. Rezende PhD,Alberto R.M. Martinez MD, PhD,Mariana R. de Brito MD,Marcondes C. França Jr MD, PhD; Mov...
Friday, October 29, 2021

Targeting 3′ and 5′ untranslated regions with antisense oligonucleotides to stabilize frataxin mRNA and increase protein expression

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Yanjie Li, Jixue Li, Jun Wang, David R Lynch, Xiulong Shen, David R. Corey, Darshan Parekh, Balkrishen Bhat, Caroline Woo, Jonathan J Cherry...