Friday, April 1, 2022

Design Therapeutics Completes Dosing in First Patient Cohort of Phase 1 Trial of DT-216 GeneTAC™ Molecule for the Treatment of Friedreich Ataxia

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CARLSBAD, Calif., March 30, 2022 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company develo...

Reata Pharmaceuticals Completes Rolling Submission of New Drug Application for Omaveloxolone for the Treatment of Patients with Friedreich’s Ataxia

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PLANO, Texas--(BUSINESS WIRE)-- Reata Pharmaceuticals, Inc. (Nasdaq: RETA), (“Reata,” the “Company,” “our,” “us,” or “we”), a clinical-stage...
Tuesday, March 29, 2022

Stealth Bio's Elamipretide Gets Orphan Drug Designation From FDA For Friedreich's Ataxia

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BOSTON ,  March 28, 2022  /PRNewswire/ -- Stealth BioTherapeutics Corp (Nasdaq:MITO), a clinical-stage biotechnology company focused on the ...
Thursday, March 24, 2022

Cerebellar Pathology in an Inducible Mouse Model of Friedreich Ataxia

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Mercado-Ayón E, Warren N, Halawani S, Rodden LN, Ngaba L, Dong YN, Chang JC, Fonck C, Mavilio F, Lynch DR and Lin H (2022). Front. Neurosci....

The U.S. Food and Drug Administration granted orphan drug designation to elamipretide to treat Friedreich’s ataxia (FRDA).

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  U.S. Food and Drug Administration,  03/22/2022   Generic Name:  elamipretide Date Designated:  03/22/2022 Orphan Designation:  Treatment o...
Wednesday, March 23, 2022

Quantitative susceptibility mapping reveals alterations of dentate nuclei in common types of degenerative cerebellar ataxias

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Deistung A, Jäschke D, Draganova R, Pfaffenrot V, Hulst T, Steiner KM, Thieme A, Giordano IA, Klockgether T, Tunc S, Münchau A, Minnerop M, ...
Friday, March 18, 2022

Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia

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ClinicalTrials.gov Identifier: NCT05285540. First Posted : March 17, 2022  The purpose of this study Phase 1 is to evaluate the safety, tol...
Thursday, March 17, 2022

Modelling Protein Plasticity: The Example of Frataxin and Its Variants

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Botticelli, S.; La Penna, G.; Nobili, G.; Rossi, G.; Stellato, F.; Morante, S.; Molecules 2022, 27, 1955. doi:10.3390/molecules27061955  Fra...
Wednesday, March 16, 2022

Control of arm movements in Friedreich's ataxia patients: role of sensory feedback

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Zhang L, Straube A, Eggert T. ; Exp Brain Res. 2022 Mar 14. doi: 10.1007/s00221-022-06343-5. Epub ahead of print. PMID: 35286422.  The model...

Difficulties translating antisense-mediated activation of Frataxin expression from cell culture to mice

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Audrius Kilikevicius, Jun Wang, Xiulong Shen, Frank Rigo, Thahza P. Prakash, Marek Napierala & David R. Corey (2022); RNA Biology, 19:1,...