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,...

Crosstalk between nucleus and mitochondria in human disease: Mitochondrial iron and calcium homeostasis in Friedreich ataxia

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Jordi Tamarit, Elena Britti, Fabien Delaspre, Marta Medina‐Carbonero, Arabela Sanz‐Alcázar, Elisa Cabiscol, Joaquim Ros; IUBMB Life. 2021; 1...
Sunday, March 7, 2021

Preclinical autoimmune disease biotech Design Therapeutics files for a $100 million IPO

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March 5, 2021, Design Therapeutics is a preclinical-stage biopharmaceutical company pioneering novel small-molecule therapeutic candidates, ...