Monday, August 8, 2016

Long-Axis Left Ventricular and Left Atrial Dysfunction in Friedreich Ataxia with Normal Ejection Fraction – Global Longitudinal Strain Versus Tissue Doppler Imaging Velocities

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D. Jackson, R. Hassam, L. Donelan, R. Peverill, Heart, Lung and Circulation, Volume 25, Supplement 2, August 2016, Page S80, ISSN 1443-9506,...
Sunday, August 7, 2016

Translating HDAC inhibitors in Friedreich’s ataxia

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Elisabetta Soragni & Joel M. Gottesfeld. Expert Opinion on Orphan Drugs, Published online: 31 Jul 2016 DOI:10.1080/21678707.2016.1215910...
Saturday, August 6, 2016

Voice in Friedreich Ataxia

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Adam P. Vogel, Mayumi I. Wardrop, Joanne E. Folker, Matthis Synofzik, Louise A. Corben, Martin B. Delatycki, Shaheen N. Awan, Journal of Voi...
Friday, August 5, 2016

The Pediatric Cerebellum in Inherited Neurodegenerative Disorders: A Pattern-recognition Approach

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Susan I. Blaser, Maja Steinlin, Almundher Al-Maawali, Grace Yoon, Neuroimaging Clinics of North America, Volume 26, Issue 3, August 2016, Pa...
Thursday, August 4, 2016

Long-term treatment with thiamine as possible medical therapy for Friedreich ataxia

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Antonio Costantini, Tiziana Laureti, Maria Immacolata Pala, Marco Colangeli, Simona Cavalieri, Elisa Pozzi, Alfredo Brusco, Sandro Salvarani...
Wednesday, August 3, 2016

Agilis Biotherapeutics Announces FDA Orphan Drug Designation for the Treatment of Friedreich’s Ataxia (FA)

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August 02, 2016 08:30 AM Eastern Daylight Time. August 02, 2016. First Gene Therapy Candidate to Receive Designation for FA. CAMBRIDGE, ...
Tuesday, August 2, 2016

Pfizer buys gene therapy company - Is it a good investment?

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Rare Disease Report. James Radke, PhD, Published Online: Monday, Aug 01, 2016 Pfizer has acquired gene therapy company Bamboo Therapeutics...
Monday, August 1, 2016

Recent advances in understanding transcription termination by RNA polymerase II.

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Travis J. Loya and Daniel Reinesa,  F1000Research 2016, 5 (F1000 Faculty Rev):1478  doi: 10.12688/f1000research.8455.1 R-loop-mediated t...
Sunday, July 31, 2016

Keys to overcoming the challenge of diagnosing autosomal recessive spinocerebellar ataxia / Claves para afrontar el reto diagnóstico de las heredoataxias recesivas

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M. Arias, Neurología, Available online 25 July 2016, ISSN 0213-4853, doi:10.1016/j.nrl.2016.06.006 Una cuidadosa evaluación clínica, acom...
Friday, July 29, 2016

OCT in Central Nervous System Diseases

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Editors: Grzybowski, Andrzej, Barboni, Piero (Eds.) SPRINGER, ISBN 978-3-319-24085-5 The disorders considered include multiple sclerosis, ...