Friedreich Ataxia Scientific News

Friedreich Ataxia and close related scientific news. Topics related to rare diseases.

Sunday, March 21, 2021

The responsiveness of gait and balance outcomes to disease progression in Friedreich ataxia

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Sarah C Milne, Seok Hun Kim, Anna Murphy, Jane Larkindale, Jennifer Farmer, Ritchie Malapira, Mary Danoudis, Jessica Shaw, Tyagi Ramakrishna...
Saturday, March 20, 2021

In vivo survival and differentiation of Friedreich ataxia iPSC-derived sensory neurons transplanted in the adult dorsal root ganglia

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Viventi S, Frausin S, Howden SE, Lim SY, Finol-Urdaneta RK, McArthur JR, Abu-Bonsrah KD, Ng W, Ivanusic J, Thompson L, Dottori M.; Stem Cell...
Friday, March 19, 2021

Research priorities for rare neurological diseases: a representative view of patient representatives and healthcare professionals from the European Reference Network for Rare Neurological Diseases

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Annemarie E. M. Post, Thomas Klockgether, G. Bernhard Landwehrmeyer, Massimo Pandolfo, Astri Arnesen, Carola Reinhard & Holm Graessner. ...
Thursday, March 18, 2021

Evaluation of the Effects of Calcitriol's in the Neurological Symptoms of Friedreich's Ataxia Patients (Calcitriol-FA)

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ClinicalTrials.gov Identifier: NCT04801303.  Recruitment Status : Not yet recruiting, First Posted : March 17, 2021, Last Update Posted ...
Sunday, March 14, 2021

The Complex Genetic Landscape of Hereditary Ataxias in Turkey and Implications in Clinical Practice

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ural, A., Şimşir, G., Tekgül, Ş., Koçoğlu, C., Akçimen, F., Kartal, E., Şen, N.E., Lahut, S., Ömür, Ö., Saner, N., Gül, T., Bayraktar, E., P...

Early onset ataxia with Marfanoid features a new varient of Friedreich s ataxia

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Khichar S.; IP Indian J Neurosci 2021;7(1):95-96, doi:10.18231/j.ijn.2021.015 A young male with ataxia since early childhood with Marfanoid...
Friday, March 12, 2021

Design Therapeutics, Inc. (DSGN)

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12/3/21. Design Therapeutics is a preclinical-stage biopharmaceutical company pioneering novel small-molecule therapeutic candidates, called...
Wednesday, March 10, 2021

Cardiomyopathy of Friedreich's Disease. Modern Methods of Diagnostic

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Fomicheva E.I., Myasnikov R.P., Selivyorstov Y.A., Illarioshkin S.N., Dadali E.L., Drapkina O.M. ; Rational Pharmacotherapy in Cardiology. 2...
Monday, March 8, 2021

Coenzyme Q10 Analogues: Benefits and Challenges for Therapeutics

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Suárez-Rivero JM, Pastor-Maldonado CJ, Povea-Cabello S, Álvarez-Córdoba M, Villalón-García I, Munuera-Cabeza M, Suárez-Carrillo A, Talaverón...

Friedreich ataxia in a family from Mali, West Africa

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heick Abdel Kader Cissé,Lassana Cissé,Hamidou O Bah,Oumar Samassékou,Assiatou Simaga,Abdoulaye Tamega,Salimata Diarra,Seybou Hassane Diallo,...
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