Sunday, October 8, 2023

Vestibular Pathology as Early Finding of Friedreich’s Ataxia in a 16 Years Old Woman

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Fernández, A.G. Vestibular Pathology as Early Finding of Friedreich’s Ataxia in a 16 Years Old Woman. Indian J Otolaryngol Head Neck Surg (2...
Wednesday, October 4, 2023

Social cognition in degenerative cerebellar ataxias

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Simona Karamazovova, Veronika Matuskova, Natalie Svecova, Martin Vyhnalek, Social cognition in degenerative cerebellar ataxias, Current Opin...

FDA Clears Exploration Trial for Higher Dose of CTI-1601 for Friedreich Ataxia

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October 2, 2023. After the company released positive phase 2 data earlier this year, Larimar Therapeutics announced that the FDA has cleared...
Sunday, October 1, 2023

Neurologic orphan diseases: Emerging innovations and role for genetic treatments

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Kioutchoukova IP, Foster DT, Thakkar RN, Foreman MA, Burgess BJ, Toms RM, Molina Valero EE, Lucke-Wold B. Neurologic orphan diseases: Emergi...

Benefits of Adaptive Sport on Physical and Mental Quality of Life in People with Physical Disabilities: A Meta-Analysis

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Isidoro-Cabañas E, Soto-Rodríguez F, Morales-Rodríguez F, Pérez-Mármol J. Benefits of Adaptive Sport on Physical and Mental Quality of Life ...

Comparison of Live and Remote Video Ratings of the Scale for Assessment and Rating of Ataxia

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Taheri Amin A, Faber J, Önder D, et al. Comparison of Live and Remote Video Ratings of the Scale for Assessment and Rating of Ataxia; Moveme...

Pre-Validation of a Virtual Reality Tool to Quantify the Severity of Friedreich's Ataxia

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K. Chenier, A. Duquette, L. Touma, M. T. Le and D. R. Labbe, "Pre-Validation of a Virtual Reality Tool to Quantify the Severity of Frie...
Thursday, September 28, 2023

CRISPR/Cas9-Based Edition of Frataxin Gene in Dictyostelium discoideum

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Hernan Gustavo Gentili, María Florencia Pignataro, Justo Facundo Olmos, María Florencia Paván, Lorena Itatí Ibáñez, Javier Santos, Francisco...
Wednesday, September 27, 2023

Long non-coding RNA TUG1 is down-regulated in Friedreich's ataxia

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Long non-coding RNA TUG1 is down-regulated in Friedreich's ataxia. Mert Koka, Hui Li, Rumana Akther, Susan Perlman, Darice Wong, Brent L...
Monday, September 25, 2023

Innovative thinking of clinical investigation for rare disease drug development

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Wang, P., Chow, SC. Innovative thinking of clinical investigation for rare disease drug development. Orphanet J Rare Dis 18, 299 (2023). doi...