Thursday, February 25, 2021

Retrotope Granted Rare Pediatric Disease Designation from FDA for Lead Development Candidate, RT001, in Two Life-Threatening Neurodegenerative Indications

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LOS ALTOS, Calif., Feb. 25, 2021 (GLOBE NEWSWIRE) -- Retrotope, a clinical-stage biopharmaceutical company focused on the development of nov...
Wednesday, February 24, 2021

Modifiers of Somatic Repeat Instability in Mouse Models of Friedreich Ataxia and the Fragile X-Related Disorders: Implications for the Mechanism of Somatic Expansion in Huntington's Disease

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Zhao X, Kumari D, Miller CJ, Kim GY, Hayward B, Vitalo AG, Pinto RM, Usdin K.; J Huntingtons Dis. 2021;10(1):149-163. doi: 10.3233/JHD-20042...

Nuclear Factor Erythroid 2-Related Factor 2 Activation Might Mitigate Clinical Symptoms in Friedreich’s Ataxia: Clues of an “Out-Brain Origin” of the Disease From a Family Study

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Petrillo S, Santoro M, La Rosa P, Perna A, Gallo MG, Bertini ES, Silvestri G and Piemonte F; Front. Neurosci. 15:638810. (2021) doi:10.3389/...
Sunday, February 21, 2021

Frataxins Emerge as New Players of the Intracellular Antioxidant Machinery

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Uceda, A.B.; Donoso, J.; Frau, J.; Vilanova, B.; Adrover, M.; Antioxidants 2021, 10, 315. doi:10.3390/antiox10020315  Consequently, our work...
Saturday, February 20, 2021

Energy deficit-independent stress response in the Frataxin-depleted heart: evidence that Integrated Stress Response can predominate over mTORC1 activation

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Cesar Vasquez, Monika Patel, Aishwarya Sivaramakrishnan, Carmen Bekeova, Lauren Anderson-Pullinger, Nadan Wang, Hsin-Yao Tang, Erin L Seifer...
Friday, February 19, 2021

Friedreich’s Ataxia and Auditory Processing Disorder

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Teive, H.A., Iliadou, V.M. & Manto, M.; Cerebellum (2021). doi:10.1007/s12311-021-01238-7 The link between auditory deprivation (in this...

Auditory Phenotypic Variability in Friedreich’s Ataxia Patients

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Koohi, N., Thomas-Black, G., Giunti, P., Doris-Eva Bamiou.; Cerebellum (2021). doi:10.1007/s12311-021-01236-9 To our knowledge, this is the ...
Thursday, February 18, 2021

Hereditary Ataxia: A Focus on Heme Metabolism and Fe-S Cluster Biogenesis

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Chiabrando D, Bertino F, Tolosano E.; Int J Mol Sci. 2020 May 26;21(11):3760. doi: 10.3390/ijms21113760. Mutations in genes involved in heme...
Sunday, February 14, 2021

Prognostic value of longitudinal strain and ejection fraction in Friedreich ataxia

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L. Legrand, C. Heuze, A. Diallo, M.L. Monin, C. Ewenczyk, E. Vicaut, G. Montalescot, R. Isnard, A. Durr, F. Pousset. International Journal o...
Friday, February 12, 2021

Data Readout from Potentially Pivotal Phase 2/3 INAD Trial Expected 1H ‘01; Phase 2 ALS and Pivotal Phase 2/3 Friedreich’s Ataxia Trial Readouts Expected by Year-End

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LOS ALTOS, Calif., Feb. 11, 2021 (GLOBE NEWSWIRE)​. RT001 Clinical Program Highlights: Friedreich’s Ataxia (FA) – Enrollment was completed i...