Wednesday, September 8, 2021

A translational approach to determine target concentrations of MIN-102 (leriglitazone) to support a Phase II study in Friedreich’s ataxia

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S. Poli, L. Rodríguez-Pascau, J. Ros, P. González-Cabo, E. Britti, D. Lynch, G. Pina, S. Pascual, M. Cerrada-Gimenez, D. Eckland, E. Traver,...
Friday, September 3, 2021

Mechanisms of impaired mitochondrial homeostasis and NAD+ metabolism in a model of mitochondrial heart disease exhibiting redox active iron accumulation

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Chiang S, Braidy N, Maleki S, Lal S, Richardson DR, Huang ML.; Redox Biol. 2021 Jun 10;46:102038. doi: 10.1016/j.redox.2021.102038. Epub ahe...
Thursday, September 2, 2021

Retrotope eyes $100m raise for neurodegenerative disease platform

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https://www.clinicaltrialsarena.com   27 Aug 2021 California-based Retrotope aims to expand its investor pool as it prepares to potentially...
Wednesday, September 1, 2021

Determine Range of Tissue Frataxin Concentrations and Other Potential Biomarkers

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ClinicalTrials.gov Identifier: NCT05028764, First Posted : August 31, 2021; Sponsor: Larimar Therapeutics, Inc.  Brief Summary: To examine ...
Sunday, August 29, 2021

Brain Structure and Degeneration Staging in Friedreich Ataxia: MRI Volumetrics from the ENIGMA-Ataxia Working Group

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Harding IH, Chopra S, Arrigoni F, Boesch S, Brunetti A, Cocozza S, Corben LA, Deistung A, Delatycki M, Diciotti S, Dogan I, Evangelisti S, F...
Tuesday, August 24, 2021

Treatment with ROS detoxifying gold quantum clusters alleviates the functional decline in a mouse model of Friedreich ataxia

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CHIARA VILLA, MARIELLA LEGATO, ALESSANDRO UMBACH, CHIARA RIGANTI, REBECCA JONES, BEATRICE MARTINI, MARINA BOIDO, CLAUDIO MEDANA, IRENE FACCH...
Monday, August 23, 2021

Data-derived wearable digital biomarkers predict Frataxin gene expression levels and longitudinal disease progression in Friedreich’s Ataxia

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Aldo Faisal, Balasundaram Kadirvelu, Constantinos Gavriel, Sathiji Nageshwaran, Ping Kei Jackson Chan, Stavros Athanasopoulos, Paola Giunti,...
Sunday, August 15, 2021

SS-31 efficacy in a mouse model of Friedreich ataxia by upregulation of frataxin expression

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Liu Y, Cai J, Shen J, Dong W, Xu L, Fang M, Lin Y, Liu J, Ding Y, Qiao T, Li K.; Hum Mol Genet. 2021 Aug 13:ddab232. doi: 10.1093/hmg/ddab2...
Saturday, August 14, 2021

CRISPR/Cas9 gene-edited HSPCs for Friedreich’s ataxia

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Stephanie Cherqui, PhD, University of California, San Diego, La Jolla, CA, describes a CRISPR/Cas9 gene-edited hematopoietic stem and progen...
Friday, August 13, 2021

Exicure, Inc. Reports Second Quarter 2021 Financial Results and Corporate Progress

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CHICAGO & CAMBRIDGE, Mass.--(BUSINESS WIRE)​; XCUR-FXN  – Friedreich’s Ataxia   The Company hosted a virtual R&D Day on July 15, 202...