Monday, August 28, 2023

Sarepta Therapeutics to Collaborate With Lexeo on Heart Disease Gene Therapy Pipeline

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Aug 28, 2023, NEW YORK – Sarepta Therapeutics has invested an undisclosed sum in Lexeo Therapeutics, and the two companies on Monday said th...
Friday, August 25, 2023

Halogens engineering-based design of agonists for boosting expression of frataxin protein in Friedreich's ataxia

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Naveed, M., Ali, I., Aziz, T., Ain, N., Shabbir, M. A., Javed, K., Alharbi, M., Alshammari, A., Alasmari, A. F., Alharbi, S. A., & Alhar...
Thursday, August 24, 2023

Design Therapeutics Reports Initial Results from Phase 1 Multiple-Ascending Dose Study of DT-216 for the Treatment of Friedrich Ataxia

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CARLSBAD, Calif., Aug. 14, 2023 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company develop...
Monday, August 21, 2023

Epidemiological research on rare diseases using large-scale online search queries and reported case data

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Zhang, L., Jin, Y., Li, J. et al. Epidemiological research on rare diseases using large-scale online search queries and reported case data. ...
Saturday, August 19, 2023

Finding an Appropriate Mouse Model to Study the Impact of a Treatment for Friedreich Ataxia on the Behavioral Phenotype

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Bouchard, C.; Gérard, C.; Yanyabé, S.G.-f.; Majeau, N.; Aloui, M.; Buisson, G.; Yameogo, P.; Couture, V.; Tremblay, J.P. Finding an Appropri...
Wednesday, August 16, 2023

Why Is Design Therapeutics Stock Moving Lower Today?

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Aug. 15, 2023, Design Therapeutics planned to reformulate its Friedreich ataxia drug following injection site reactions in a Phase 1 study. ...

Rationale and protocol of a double-blind, randomized, placebo-controlled trial to test the efficacy, safety and tolerability of Dimethyl Fumarate in Friedreich Ataxia (DMF-FA-201)

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Chiara Pane1, Alberto M. Marra, Ludovica Aliberti, Mario Campanile, Federica Coscetta, Giulia Crisci, Roberta D'Assante, Angela Marsili,...

Interferon Gamma Enhances Cytoprotective Pathways via Nrf2 and MnSOD Induction in Friedreich’s Ataxia Cells

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Luffarelli, R.; Panarello, L.; Quatrana, A.; Tiano, F.; Fortuni, S.; Rufini, A.; Malisan, F.; Testi, R.; Condò, I. Interferon Gamma Enhances...
Thursday, August 10, 2023

Omaveloxolone for the Treatment of Friedreich’s Ataxia

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Riley Kessler, Sonal Sharma, David R Lynch, Omaveloxolone for the Treatment of Friedreich’s Ataxia, Published Online: Aug 9th 2023 touchREVI...
Monday, August 7, 2023

CRISPR/Cas9 Genome Editing for Tissue-Specific In Vivo Targeting: Nanomaterials and Translational Perspective

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Sahel DK, Vora LK, Saraswat A, et al. CRISPR/Cas9 Genome Editing for Tissue-Specific In Vivo Targeting: Nanomaterials and Translational Pers...