Saturday, October 5, 2019

SINEUP non-coding RNAs rescue defective frataxin expression and activity in a cellular model of Friedreich's Ataxia

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Carlotta Bon, Riccardo Luffarelli, Roberta Russo, Silvia Fortuni, Bianca Pierattini, Chiara Santulli, Cristina Fimiani, Francesca Persichett...
Friday, October 4, 2019

A case of Friedreich ataxia in an adolescent with 16p11.2 microdeletion syndrome

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Valentina Pelliccia, Silvia Ferranti, Rosa Mostardini, Salvatore Grosso; Neurol Sci (2019). doi:10.1007/s10072-019-04075-z A completely di...
Monday, September 30, 2019

Health-related quality of life and depressive symptoms in Friedreich ataxia

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Javier Pérez-Flores, Atteneri Hernández-Torres, Fernando Montón, Antonieta Nieto; Qual Life Res. 2019 Sep 28. doi: 10.1007/s11136-019-02311-...
Friday, September 27, 2019

Physicochemical Characterization and Antioxidant Activity Evaluation of Idebenone/Hydroxypropyl-β-Cyclodextrin Inclusion Complex

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Venuti, V.; Crupi, V.; Fazio, B.; Majolino, D.; Acri, G.; Testagrossa, B.; Stancanelli, R.; De Gaetano, F.; Gagliardi, A.; Paolino, D.; Flor...
Thursday, September 26, 2019

A Randomized, Double-Blind, Controlled, Phase 2/3 Study to Assess Efficacy, Long Term Safety and Tolerability of RT001 in Subjects With Friedreich's Ataxia

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ClinicalTrials.gov Identifier: NCT04102501, September 25, 2019. Retrotope, Inc, drug RT001 (9-cis, 12-cis-11,11-D2-linoleic acid ethyl es...
Tuesday, September 24, 2019

Targeting BDNF signaling by natural products: novel synaptic repair therapeutics for neurodegeneration and behavior disorders

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Sweta Bawari, Devesh Tewari, Sandro Argüelles, Archana N. Sah, Seyed Fazel Nabavi, Suowen Xu, Rosa Anna Vacca, Seyed Mohammad Nabavi, Samira...
Friday, September 20, 2019

The CureFA Foundation, Rye, NY, USA, has joined the ownership of Fratagene Therapeutics

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Fratagene Therapeutics. September 2019. The CureFA Foundation, Rye, NY, USA, has joined the ownership of Fratagene Therapeutics. The CureFA...
Friday, September 13, 2019

Mechanism of activation of the human cysteine desulfurase complex by frataxin

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Shachin Patra and David P. Barondeau; PNAS first published September 11, 2019 doi:10.1073/pnas.1909535116 Our results support key roles fo...
Thursday, September 12, 2019

Two new Pfizer-coauthored studies validate Novoheart’s pioneering human bioengineered heart tissues and chambers for improving drug development

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Novoheart Holdings Inc.; September 09, 2019. Novoheart seeks to revolutionize human heart tissue engineering for disease modeling and drug...
Tuesday, September 10, 2019

Changes detected in swallowing function in Friedreich ataxia over 12 months

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Megan Keage, Martin B. Delatycki, Jessamy Dyer, Louise A. Corben, Adam P. Vogel, Neuromuscular Disorders, 2019, doi:10.1016/j.nmd.2019.08.01...