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...

Future Prospects of Gene Therapy for Friedreich’s Ataxia

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Ocana-Santero, G.; Díaz-Nido, J.; Herranz-Martín, S.; Int. J. Mol. Sci. 2021, 22, 1815. doi:10.3390/ijms22041815  Friedreich’s ataxia is an ...
Monday, February 8, 2021

Sheffield university team awarded £1.6m for drug research​​​​​​​ on motor neurone disease

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Sheffield Telegraph; It will support their partnership with Aclipse Therapeutics to advance the translational development of M102 - a drug c...
Sunday, February 7, 2021

Gauging Gait Disorders with a Method Inspired by Motor Control Theories: A Pilot Study in Friedreich’s Ataxia

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Gouelle, A.; Norman, S.; Sharot, B.; Salabarria, S.; Subramony, S.; Corti, M. Gauging; Sensors 2021, 21, 1144. doi:10.3390/s21041144   Organ...
Wednesday, February 3, 2021

Defective palmitoylation of transferrin receptor triggers iron overload in Friedreich's ataxia fibroblasts

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Floriane Petit, Anthony Drecourt, Michaël Dussiot, Coralie Zangarelli, Olivier Hermine, Arnold Munnich, Agnes Rotig; Blood blood.2020006987....
Sunday, January 31, 2021

Therapeutic roles of natural remedies in combating hereditary ataxia: A systematic review

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Michael Weng Lok Phang, Sze Yuen Lew, Ivy Chung, William Kiong-Seng Lim, Lee Wei Lim & Kah Hui Wong; Chin Med 16, 15 (2021). https://doi...
Saturday, January 30, 2021

Replication-independent instability of Friedreich's ataxia GAA repeats during chronological aging

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Neil AJ, Hisey JA, Quasem I, McGinty RJ, Hitczenko M, Khristich AN, Mirkin SM.; Proc Natl Acad Sci U S A. 2021 Feb 2;118(5):e2013080118. do...
Tuesday, January 26, 2021

Ectopic Burden Via Holter Monitors in Friedreich’s Ataxia,

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Erika Mejia, Abigail Lynch, Patrick Hearle, Oluwatimilehin Okunowo, Heather Griffis, Maully Shah, David Lynch, Kimberly Y. Lin; Pediatric Ne...
Monday, January 25, 2021

AVXS-401, by Novartis Gene Therapies

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25/01/2021. AVXS-401 is a gene therapy developed by Novartis Gene Therapies (AveXis, now is part of Novartis). It uses the AAV9 adeno-associ...