Wednesday, December 27, 2023

Omaveloxolone approved for patients aged 16 years and older with Friedreich ataxia (FRDA): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)

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Arthur Lenahan, Sho Yano, Brett Graham, Kuntal Sen, Omaveloxolone approved for patients aged 16 years and older with Friedreich ataxia (FRDA...
Tuesday, December 26, 2023

CO105 A Retrospective Study Characterizing Age at Loss of Ambulation Among Patients With Friedreich Ataxia Using Health Administrative Claims Data in the United States

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A. Salvucci, C. Qian, L. Powell, D. Lynch, G. Vasco, K. Johnston, I. Tomazos, CO105 A Retrospective Study Characterizing Age at Loss of Ambu...

Mitochondrial impairment, decreased sirtuin activity and protein acetylation in dorsal root ganglia in Friedreich Ataxia models

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Arabela Sanz-Alcázar, Elena Britti, Fabien Delaspre, Marta Medina-Carbonero, Maria Pazos-Gil, Jordi Tamarit, Joaquim Ros & Elisa Cabisco...

Predictors of Survival in Friedreich's Ataxia: A Prospective Cohort Study

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Indelicato, E., Reetz, K., Maier, S., Nachbauer, W., Amprosi, M., Giunti, P., Mariotti, C., Durr, A., de Rivera Garrido, F.J.R., Klopstock, ...

Generation and characterization of two human iPSC lines, IGIBi014-A and IGIBi015-A, from Friedreich's ataxia (FRDA) patients with pathogenic (GAA/TTC)n repeat expansion in first intron of the Frataxin (FXN) gene

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Ahmad I, Kapoor H, Kumar Srivastava A, Faruq M. Generation and characterization of two human iPSC lines, IGIBi014-A and IGIBi015-A, from Fri...
Saturday, December 23, 2023

Loss of filamentous actin, tight junction protein expression, and paracellular barrier integrity in Frataxin-deficient human brain microvascular endothelial cells -implications for blood-brain barrier physiology in Friedreich's Ataxia

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Frances M. SMITH, Daniel Kosman; Front. Mol. Biosci. Sec. Lipids, Membranes and Membranous Organelles, Volume 10 - 2023 | doi:10.3389/fmolb....
Friday, December 22, 2023

EFICACIA Y SEGURIDAD DE LA OMAVELOXOLONA EN LA ATAXIA DE FRIEDREICH

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Resumen -SIIC  en castellano:   Disorders 38(2):313-320. Autores: Lynch D R, Chin MP, Meyer CJ  Filadelfia, EE.UU. En pacientes con ataxia d...

JNS-101 by Jupiter Neurosciences for Friedreich Ataxia: Likelihood of Approval

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December 21, 2023. JNS-101 is under clinical development by Jupiter Neurosciences and currently in Phase II for Friedreich Ataxia. According...

MIB-626 by Metro International Biotech for Friedreich Ataxia: Likelihood of Approval

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December 21, 2023. MIB-626 is under clinical development by Metro International Biotech and currently in Phase II for Friedreich Ataxia. Acc...
Friday, December 15, 2023

Skyclarys* (omaveloxolone) received a positive opinion from the CHMP for the treatment of Friedreich’s ataxia

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Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 11-14 December 2023. Skyclarys* (omaveloxolone) received a...