Monday, February 28, 2022

Design Therapeutics Announces FDA Clearance of Investigational New Drug Application for First GeneTAC™ Molecule for Friedreich Ataxia

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CARLSBAD, Calif., Feb. 28, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments ...

Accurate and simple FXN-GAA repeats (Friedreich ataxia loci) estimation by long read targeted sequencing.

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Pooja Sharma, Bharathram Uppilli, Istaq Ahmad, Shweta Sahni, Mohammed Faruq; bioRxiv 2022.02.24.481841; doi:10.1101/2022.02.24.481841   We d...
Sunday, February 27, 2022

Mitochondrial De Novo Assembly of Iron–Sulfur Clusters in Mammals: Complex Matters in a Complex That Matters

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Citation: Perfitt, T.L.; Martelli, A.; Inorganics 2022, 10, 31. doi:10.3390/inorganics10030031 Recently obtained structural and biochemical ...
Saturday, February 26, 2022

Selected Histone Deacetylase Inhibitors Reverse the Frataxin Transcriptional Defect in a Novel Friedreich’s Ataxia Induced Pluripotent Stem Cell-Derived Neuronal Reporter System

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Schreiber Anna M., Li Yanjie, Chen Yi-Hsien, Napierala Jill S., Napierala Marek; Front. Neurosci., 16, 23 February 2022 doi:10.3389/fnins.20...
Thursday, February 24, 2022

Jupiter Neurosciences, Inc. Announces Completion of Phase I Safety Trial with Enhanced Resveratrol Product, JOTROL™ Jupiter Neurosciences logo

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JUPITER, Fla. and BOSTON, Feb. 23, 2022 /PRNewswire/ -- Jupiter Neurosciences, Inc. ("Jupiter" or the "Company"), today ...
Thursday, February 17, 2022

Downregulation of Three Immune-Specific Core Genes and the Regulatory Pathways in Children and Adult Friedreich's Ataxia: A Comprehensive Analysis Based on Microarray

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Liu, Lichun; Lai, Yongxing; Zhan, Zhidong; Fu, Qingxian; Jiang, Yuelian (2022). Front. Neurol., 14 February 2022, doi:10.3389/fneur.2021.816...

Selection of Synthetic Proteins to Modulate the Human Frataxin Function

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Pignataro MF, Herrera MG, Fernández NB, Aran M, Bataglini F, Santos J; Preprint from bioRxiv, 12 Feb 2022, DOI: 10.1101/2022.02.11.480108   ...
Wednesday, February 16, 2022

LEXEO Therapeutics Announces FDA Clearance of Investigational New Drug Application for LX2006, an AAV-based Gene Therapy Candidate for Friedreich’s Ataxia Cardiomyopathy

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NEW YORK, Feb. 16, 2022 (GLOBE NEWSWIRE) -- LEXEO Therapeutics. LEXEO today announced that the U.S. Food and Drug Administration (FDA) has c...

FDA Maintains Clinical Hold on Larimar’s Friedreich’s Ataxia Study

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Global Genes. February 15, 2022. Larimar Therapeutics, a company focused on developing treatments for complex rare diseases, said that the U...
Tuesday, February 15, 2022

Retrotope announces Phase 2/3 trial of RT001 in FA did not successfully meet its endpoints

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Friedreich's Ataxia Research Alliance, 14 February 2022.  FARA has been notified by Retrotope that the recently completed Phase 2/3 tria...