Friedreich Ataxia Scientific News

Friedreich Ataxia and close related scientific news. Topics related to rare diseases.

Saturday, September 2, 2023

Proprioceptive and tactile processing in individuals with Friedreich Ataxia, an fMRI study

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Destrebecq Virginie, Rovai Antonin, Trotta Nicola, Comet Camille, Gilles Naeije, Front. Neurol. Sec. Movement Disorders, Volume 14 - 2023, ...
Thursday, August 31, 2023

SIRT3 Regulates Clearance of Apoptotic Cardiomyocytes by Deacetylating Frataxin

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Jing Gao, Chenglin Huang, Linghui Kong, Wugang Zhou, Mengwei Sun, Tong Wei and Weili Shen. Circulation Research. 2023;0. Originally pu...

Replication Stalling at Friedreich's Ataxia (GAA)n Repeats In Vivo

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Maria M. Krasilnikova & Sergei M. Mirkin (2004) Replication Stalling at Friedreich's Ataxia (GAA)n Repeats In Vivo, Molecular and Ce...
Tuesday, August 29, 2023

Longitudinal changes of SARA scale in Friedreich ataxia: Strong influence of baseline score and age at onset

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Porcu, L., Fichera, M., Nanetti, L., Rulli, E., Giunti, P., Parkinson, M.H., Durr, A., Ewenczyk, C., Boesch, S., Nachbauer, W., Indelicato, ...
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. ...
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Juan Carlos Baiges
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