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...

Nrf2 Induction Re-establishes a Proper Neuronal Differentiation Program in Friedreich’s Ataxia Neural Stem Cells

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La Rosa Piergiorgio, Russo Marta, D’Amico Jessica, Petrillo Sara, Aquilano Katia, Lettieri-Barbato Daniele, Turchi Riccardo, Bertini Enrico ...
Tuesday, July 30, 2019

The neuroprotective mechanisms and effects of sulforaphane

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Klomparens EA, Ding Y.; Brain Circ 2019;5:74-83 DOI: 10.4103/bc.bc_7_19 Sulforaphane (SFN) is a phytochemical found in cruciferous vegetab...
Monday, July 29, 2019

Yeast as a Tool for Deeper Understanding of Human Manganese-Related Diseases

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Louise Thines, Antoine Deschamps, Jiri Stribny and Pierre Morsomme; Genes 2019, 10(7), 545; (Review) doi:10.3390/genes10070545 Several ma...
Saturday, July 27, 2019

The path toward using microbial metabolites as therapies

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Hélène C. Descamps, Beatrice Herrmann, Daphne Wiredu, Christoph A. Thaiss; EBioMedicine (Published by The Lancet), Volume 44, 2019, Pages 74...