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...
Saturday, October 23, 2021

Iron Metabolism in Pancreatic Beta-Cell Function and Dysfunction

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Marku, A.; Galli, A.; Marciani, P.; Dule, N.; Perego, C.; Castagna, M.; Cells 2021, 10, 2841. doi:10.3390/cells10112841  Iron is an essenti...

FDA refusal of Stealth Bio drug shows challenges of ultra-rare disease studies

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https://medcitynews.com The FDA refused to review Stealth BioTherapeutics’ Barth syndrome drug, telling the company results in a study of ju...

Mitochondrial Iron-Sulfur Cluster Biogenesis and Neurological Disorders

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Arthavan Selvanathan, Bindu Parayil Sankaran; Mitochondrion, 2021, doi:10.1016/j.mito.2021.10.004. This review focuses on the disorders of I...