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...
Saturday, August 7, 2021

Un bri d’esperança

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Dissabte, 7 agost 2021, El Punt Avui. L’atàxia de Friedreich provoca problemes de coordinació, força muscular i cor i escurça l’esperança de...

Digital endpoints for self-administered home-based functional assessment in pediatric Friedreich’s ataxia

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Arne Mueller, Elaine Paterson, Avery McIntosh, Jens Praestgaard, Mary Bylo, Holger Hoefling, McKenzie Wells, David R. Lynch, Christian Rumme...