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...
Friday, August 6, 2021

The Role of Voltage-Dependent Anion Channel in Mitochondrial Dysfunction and Human Disease

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Varughese J, Buchanan S, Pitt A., Cells. 2021 Jul;10(7). PMCID: PMC8305817 doi:10.3390/cells10071737 The voltage-dependent anion channel (VD...
Thursday, August 5, 2021

Determining the Validity of Conducting Rating Scales in Friedreich Ataxia through Video

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Tai, G., Corben, L.A., Woodcock, I.R., Yiu, E.M. and Delatycki, M.B. (2021); Mov Disord Clin Pract, 8: 688-693. doi:10.1002/mdc3.13204  Exce...