Monday, March 8, 2021

Coenzyme Q10 Analogues: Benefits and Challenges for Therapeutics

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Suárez-Rivero JM, Pastor-Maldonado CJ, Povea-Cabello S, Álvarez-Córdoba M, Villalón-García I, Munuera-Cabeza M, Suárez-Carrillo A, Talaverón...

Friedreich ataxia in a family from Mali, West Africa

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heick Abdel Kader Cissé,Lassana Cissé,Hamidou O Bah,Oumar Samassékou,Assiatou Simaga,Abdoulaye Tamega,Salimata Diarra,Seybou Hassane Diallo,...

Crosstalk between nucleus and mitochondria in human disease: Mitochondrial iron and calcium homeostasis in Friedreich ataxia

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Jordi Tamarit, Elena Britti, Fabien Delaspre, Marta Medina‐Carbonero, Arabela Sanz‐Alcázar, Elisa Cabiscol, Joaquim Ros; IUBMB Life. 2021; 1...
Sunday, March 7, 2021

Preclinical autoimmune disease biotech Design Therapeutics files for a $100 million IPO

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March 5, 2021, Design Therapeutics is a preclinical-stage biopharmaceutical company pioneering novel small-molecule therapeutic candidates, ...
Thursday, March 4, 2021

Ectopic Burden via Holter Monitors in Friedreich Ataxia.

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Erika Mejia,Abigail Lynch,Patrick Hearle,Oluwatimilehin Okunowo,Heather Griffis,Maully Shah,David Lynch,Kimberly Y Lin; Physician's Week...

Larimar Therapeutics Reports Fourth Quarter and Full Year 2020 Operating and Financial Results

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BioSpace.com, Published: Mar 04, 2021​.    Reported preliminary Phase 1 findings from a Single Ascending Dose (SAD) trial that suggest singl...
Wednesday, March 3, 2021

LEXEO Therapeutics Announces License Agreement and Consolidation of Comprehensive Pre-clinical Data Package to Support Cardiac Friedreich's Ataxia Gene Therapy Program (LX2006)

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Published: Mar 01, 2021 NEW YORK, March 01, 2021 (GLOBE NEWSWIRE) -- LEXEO Therapeutics, a clinical-stage gene therapy company, today annou...
Friday, February 26, 2021

The displacement of frataxin from the mitochondrial cristae correlates with abnormal respiratory supercomplexes formation and bioenergetic defects in cells of Friedreich ataxia patients

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Davide Doni, Giovanni Rigoni, Elisa Palumbo, Elisa Baschiera, Roberta Peruzzo, Edith De Rosa, Federico Caicci, Leonardo Passerini, Daniela B...
Thursday, February 25, 2021

Retrotope Granted Rare Pediatric Disease Designation from FDA for Lead Development Candidate, RT001, in Two Life-Threatening Neurodegenerative Indications

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LOS ALTOS, Calif., Feb. 25, 2021 (GLOBE NEWSWIRE) -- Retrotope, a clinical-stage biopharmaceutical company focused on the development of nov...
Wednesday, February 24, 2021

Modifiers of Somatic Repeat Instability in Mouse Models of Friedreich Ataxia and the Fragile X-Related Disorders: Implications for the Mechanism of Somatic Expansion in Huntington's Disease

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Zhao X, Kumari D, Miller CJ, Kim GY, Hayward B, Vitalo AG, Pinto RM, Usdin K.; J Huntingtons Dis. 2021;10(1):149-163. doi: 10.3233/JHD-20042...