Thursday, October 31, 2019

Potential biomarker identification for Friedreich’s ataxia using overlapping gene expression patterns in patient cells and mouse dorsal root ganglion

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Marissa Z. McMackin, Blythe Durbin-Johnson, Marek Napierala, Jill S. Napierala, Luis Ruiz, Eleonora Napoli, Susan Perlman, Cecilia Giulivi, ...
Wednesday, October 30, 2019

PTC Therapeutics Reports Third Quarter 2019 Financial Results and Provides a Corporate Update

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SOUTH PLAINFIELD, N.J., Oct. 29, 2019 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and reported ...

Symposium updates Friedreich’s ataxia community on advances against FA

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Written by Anne DeLotto Baier · October 29, 2019 @ 12:55 pm · Filed under Hot News, Morsani College of Medicine, Neurosciences & Brain R...
Sunday, October 27, 2019

Predictors of Left Ventricular Dysfunction in Friedreich’s Ataxia in a 16-Year Observational Study

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Lise Legrand, Abdourahmane Diallo, Marie-Lorraine Monin, Claire Ewenczyk, Perrine Charles, Richard Isnard, Eric Vicaut, Gilles Montalescot, ...
Saturday, October 26, 2019

Ironing the mitochondria: Relevance to its dynamics

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Mamta Upadhyay, Saloni Agarwal, Mitochondrion, 2019, doi:10.1016/j.mito.2019.09.007. The mitochondrion is “jack of many trades and master ...
Friday, October 25, 2019

Cerebellum and cognition in Friedreich ataxia: a voxel-based morphometry and volumetric MRI study

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Sirio Cocozza, Te*resa Costabile, Giuseppe Pontillo, Maria Lieto, Camilla Russo, I Leonardo Radice,Chiara Pane, Alessandro Filla, Arturo Br...
Thursday, October 24, 2019

Ethical, legal, and social issues (ELSI) in rare diseases: a landscape analysis from funders

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Adam L. Hartman, Anneliene Hechtelt Jonker, Melissa A. Parisi, Daria Julkowska, Nicole Lockhart & Rosario Isasi ; Eur J Hum Genet. 2019 ...
Wednesday, October 23, 2019

PTC Therapeutics enters collaboration with Aldevron to support GMP plasmid manufacturing

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South Plainfield, New Jersey. Thursday, October 10, 2019. PTC Therapeutics, global biopharmaceutical company, announced a collaboration wi...
Tuesday, October 22, 2019

Targeting NRF2 for the Treatment of Friedreich’s Ataxia: A Comparison among Drugs

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Petrillo, S.; D’Amico, J.; La Rosa, P.; Bertini, E.S.; Piemonte, F. Int. J. Mol. Sci. 2019, 20, 5211. Doi:10.3390/ijms20205211 (registering ...
Sunday, October 20, 2019

Safety and Biodistribution of Human Bone Marrow-Derived Mesenchymal Stromal Cells Injected Intrathecally in Non-Obese Diabetic Severe Combined Immunodeficiency Mice: Preclinical Study

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Mari Paz Quesada, David García-Bernal, Diego PastorAlicia Estirado, Miguel Blanquer, Ana Mª García-HernándezJosé, M. Moraleda, Salvador Mart...