Sunday, August 11, 2019

Exploring the Potential of Small Molecule-Based Therapeutic Approaches for Targeting Trinucleotide Repeat Disorders

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Arun Kumar Verma & Eshan Khan & Sonali R. Bhagwat & Amit Kumar; Mol Neurobiol (2019). doi:10.1007/s12035-019-01724-4 In recent...
Saturday, August 10, 2019

Excision of the expanded GAA repeats corrects cardiomyopathy phenotypes of iPSC-derived Friedreich's ataxia cardiomyocytes

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Jixue Li, Natalia Rozwadowska, Amanda Clark, Daniel Fil, Jill S. Napierala, Marek Napierala; Stem Cell Research, 2019, 101529, doi:10.1016/j...
Friday, August 9, 2019

Physiologically relevant reconstitution of iron-sulfur cluster biosynthesis uncovers persulfide-processing functions of ferredoxin-2 and frataxin

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Sylvain Gervason, Djabir Larkem, Amir Ben Mansour, Thomas Botzanowski, Christina S. Müller, Ludovic Pecqueur, Gwenaelle Le Pavec, Agnès Dela...
Thursday, August 8, 2019

A Collaborative-Filtering-Based Data Collection Strategy for Friedreich’s Ataxia

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Wenbin Yue, Zidong Wang, Bo Tian, Annette Payne, Xiaohui Liu. Cogn Comput (2019). doi:10.1007/s12559-019-09674-8 We propose a novel data c...
Wednesday, August 7, 2019

Reata gets another shot in Friedreich’s ataxia

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EvaluatePharma. August 06, 2019. Pharmaceuticals will soon find out if its decision to persevere with omaveloxolone despite an earlier fai...
Monday, August 5, 2019

Pharmaceutical compounding of orphan active ingredients in Belgium: how community and hospital pharmacists can address the needs of patients with rare diseases

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V. Vanhoorne, E. Peeters, I. Van Tongelen, K. Boussery, E. Wynendaele, B. De Spiegeleer, J. P. Remon & C. Vervaet ; Orphanet Journal of ...
Sunday, August 4, 2019

Exploring iron-binding to human frataxin and to selected Friedreich ataxia mutants by means of NMR and EPR spectroscopies

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Massimo Bellanda, Lorenzo Maso, Davide Doni, Marco Bortolus, Edith De Rosa, Federica Lunardi, Arianna Alfonsi, Martín Ezequiel Noguera, Mari...
Saturday, August 3, 2019

BioMarin announced plans to discontinue preclinical studies in the BMN290 programme for Friedreich’s Ataxia

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August 2019. BioMarin’s BMN 290 Programme: An Update for the Friedreich’s Ataxia Community On August 1, 2019 BioMarin announced plans to dis...
Friday, August 2, 2019

Low frataxin mRNA expression is associated with inflammation and oxidative stress in patients with type 2 diabetes

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Etiane Tatsch José A.M. De Carvalho Yãnaí S. Bollick Thiago Duarte Marta M.M.F. Duarte Rodrigo A. Vaucher Melissa O. Premaor Fabio V. Comim ...
Thursday, August 1, 2019

REGENXBIO Announces New License Agreement with Pfizer for the Treatment of Friedreich's Ataxia Using NAV® AAV9 Vector

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ROCKVILLE, Md., July 31, 2019 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to impro...