Monday, March 7, 2022

Gene therapy for Friedreich ataxia: Too much, too little, or just right?

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R. Mark Payne, Molecular Therapy - Methods & Clinical Development, Volume 25, 2022, Pages 1-2, doi:10.1016/j.omtm.2022.02.008  Defining ...

1st Hellenic Friedreich's Ataxia Conference

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7.3.2022; The Hellenic Friedreich's Ataxia Association plans to conduct on 6th May the 1st Hellenic Friedreich's Ataxia Conference, ...
Saturday, March 5, 2022

Update on Novartis Gene Therapies Friedreich’s ataxia Program

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Novartis Gene Therapies,  2022 . Novartis Gene Therapies had been developing a therapeutic approach for Friedreich’s ataxia. One candidate, ...
Thursday, March 3, 2022

Establishing and boosting communication in the European Reference Network for Rare Neurological Diseases (ERN-RND): the impact of offering free educational webinars

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Alicia Brunelle Praschberger, Annemarie E. M. Post, Sanja Hermanns & Holm Graessner; Orphanet J Rare Dis 17, 89 (2022). doi:10.1186/s130...

Larimar Therapeutics Inc. (NASDAQ: LRMR) Announces That The FDA Has Retained the Clinical Hold on CTI-1601

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BioPharma Journal, March 2, 2022. Larimar Therapeutics Inc. has announced that it has received feedback from the US FDA about the clinical h...

Recent Advances in the Elucidation of Frataxin Biochemical Function Open Novel Perspectives for the Treatment of Friedreich’s Ataxia

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Monfort B, Want K, Gervason S and D’Autréaux B.; Front. Neurosci. 16:838335. (2022) doi: 10.3389/fnins.2022.838335  A picture is now emergin...
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...