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...

Larimar Therapeutics Reports Second Quarter 2021 Operating and Financial Results

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BALA CYNWYD, Pa., Aug. 12, 2021 (GLOBE NEWSWIRE), “We finished the second quarter in a strong financial position and with a compelling clini...
Tuesday, August 10, 2021

Reata Pharmaceuticals, Inc. Announces Second Quarter 2021 Financial Results and Provides an Update on Clinical Development Programs

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August 09, 2021, PLANO, Texas--(BUSINESS WIRE)--Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” “our,” “us,” or “we”), a...

Design Therapeutics Reports GeneTAC™ Portfolio Progress and Second Quarter 2021 Results

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CARLSBAD, Calif., Aug. 09, 2021 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments ...
Sunday, August 8, 2021

Diagnostic and Prognostic Value of Cardiovascular Magnetic Resonance in Neuromuscular Cardiomyopathies

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Almogheer B, Antonopoulos AS, Azzu A, Al Mohdar S, Vlachopoulos C, Pantazis A, Mohiaddin RH.; Pediatr Cardiol. 2021 Aug 3. doi: 10.1007/s002...