Wednesday, March 9, 2022

The Cognitive Profile of Friedreich Ataxia: A Systemic Review and Meta-Analysis

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Naeije g, Schul JB, Corben L.; Research Square; 2022. DOI: 10.21203/rs.3.rs-1010466/v1.  Individuals with FRDA display significantly lower p...
Tuesday, March 8, 2022

Clinilabs is currently recruiting volunteers for DT-216 Clinical trial

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March 8th, 2022
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...