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...
Friday, October 18, 2019

Protocol of a randomized, double-blind, placebo-controlled, parallel-group, multicentre study of the efficacy and safety of nicotinamide in patients with Friedreich ataxia (NICOFA)

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Kathrin Reetz, Ralf-Dieter Hilgers, Susanne Isfort, Marc Dohmen, Claire Didszun, Kathrin Fedosov, Jennifer Kistermann, Caterina Mariotti, Al...
Thursday, October 17, 2019

MINORYX THERAPEUTICS RECEIVES FDA ORPHAN DRUG DESIGNATION FOR LERIGLITAZONE IN FRIEDREICH’S ATAXIA

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17 October, 2019. Mataró, Barcelona, Spain and Charleroi, Belgium, October 17, 2019 – Minoryx Therapeutics, a company that specializes in t...
Wednesday, October 16, 2019

Management Call to Discuss Positive Topline Pivotal MOXIe Data

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Reata Pharmaceuticals, OCT 2019 Management Call to Discuss Positive Topline Pivotal MOXIe Data

Orphan Drugs In Development For The Treatment Of Friedreich’s Ataxia: Focus On Omaveloxolone

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Shaila D Ghanekar, Wai Wai Miller, Colin J Meyer, Kevin J Fenelon, Alvin Lacdao, Theresa A Zesiewicz; Degenerative Neurological and Neuromus...