Tuesday, December 24, 2019

Rating scales for rare neurological diseases

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Massimo Pandolfo; Neurol Genet Dec 2019, 5 (6) e380; DOI: 10.1212/NXG.0000000000000380 While only future experience will establish whether...
Monday, December 23, 2019

Nanoscopic X‐ray imaging and quantification of the iron cellular architecture within single fibroblasts of Friedreich's ataxia patients

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De Samber, B., Vanden Berghe, T., Meul, E., Bauters, S., Seyrich, M., Smet, J., De Paepe, B., da Silva, J.C., Bohic, S., Cloetens, P., Van C...
Sunday, December 22, 2019

Age of onset determines intrinsic functional brain architecture in Friedreich ataxia

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Naeije, G., Wens, V., Coquelet, N., Sjøgård, M., Goldman, S., Pandolfo, M. and De Tiège, X.P. (2019). Ann Clin Transl Neurol. doi:10.1002/a...
Saturday, December 21, 2019

Psychometric properties of the Friedreich Ataxia Rating Scale

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Christian Rummey, Louise A. Corben, Martin B. Delatycki, S.H. Subramony, Khalaf Bushara, Christopher M. Gomez, Joseph Chad Hoyle, Grace Yoon...
Friday, December 20, 2019

A new tool to determine the cellular metabolic landscape: nanotechnology to the study of Friedreich’s ataxia

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Tommaso Vannocci, Simone Dinarelli, Marco Girasole, Annalisa Pastore & Giovanni Longo; Sci Rep 9, 19282 (2019) doi:10.1038/s41598-019-55...
Thursday, December 19, 2019

Electrophysiological evidence for limited progression of the proprioceptive impairment in Friedreich ataxia

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G. Naeije, M. Bourguignon, V. Wens, B. Marty, S. Goldman, R. Hari, V. Jousmäki, M. Pandolfo, X. De Tiège; Clinical Neurophysiology, 2019, Do...
Wednesday, December 18, 2019

Zafgen and Chondrial Therapeutics Announce Definitive Merger Agreement

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BOSTON and BALA CYNWYD, Pa., Dec. 18, 2019 (GLOBE NEWSWIRE) -- Zafgen, Inc. (ZFGN) and Chondrial Therapeutics, Inc., today announced they ha...

Chondrial Therapeutics Announces Dosing of First Patients in Phase 1 Clinical Program of CTI-1601 for Treatment of Friedreich’s AtaxiaCTI-1601 granted Rare Pediatric Disease Designation and Fast Track Designation by U.S. FDA

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BALA CYNWYD, Pa., Dec. 18, 2019 (GLOBE NEWSWIRE) -- Chondrial Therapeutics, a clinical-stage biotechnology company focused on developing tre...

Exicure : Announces First Neurological Development Program in Friedreich's Ataxia and Expands Scientific Advisory Board

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12/17/2019, CHICAGO - Exicure, Inc. (NASDAQ: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic a...
Tuesday, December 17, 2019

Cellular Stress-Modulating Drugs Can Potentially Be Identified by in Silico Screening with Connectivity Map (CMap)

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Gao Y, Sungwoo Kim, Lee YI, Lee J; International Journal of Molecular Sciences, 09 Nov 2019, 20(22) DOI: 10.3390/ijms20225601 A target...