Friday, June 16, 2023

Anodal Cerebellar Transcranial Direct Current Stimulation Reduces Motor and Cognitive Symptoms in Friedreich's Ataxia: A Randomized, Sham-Controlled Trial

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Naeije, G., Rovai, A., Destrebecq, V., Trotta, N. and De Tiège, X. (2023), Anodal Cerebellar Transcranial Direct Current Stimulation Reduces...
Tuesday, June 13, 2023

LEXEO Therapeutics Announces Completion of First Cohort and Dosing in Second Cohort in SUNRISE-FA, a Phase 1/2 Clinical Trial of LX2006 for the Treatment of Friedreich’s Ataxia Cardiomyopathy

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NEW YORK, June 13, 2023 (GLOBE NEWSWIRE) -- LEXEO Therapeutics (LEXEO), a clinical-stage gene therapy company advancing adeno-associated vir...

A Retrospective Claims Analysis Characterizing Health Care Resource Use Among Patients with Friedreich Ataxia in the United States,

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C. Qian, D. Lynch, L. Powell, A. Salvucci, G. Vasco, K.M Johnston, I. Tomazos, A Retrospective Claims Analysis Characterizing Health Care Re...

The Clinical Burden of Friedreich Ataxia: A Retrospective Claims Analysis in the United States

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L. Powell, D. Lynch, C. Qian, A. Salvucci, G. Vasco, K.M Johnston, I. Tomazos, The Clinical Burden of Friedreich Ataxia: A Retrospective Cla...
Monday, June 12, 2023

Adult-Onset Neuroepidemiology in Finland: Lessons to Learn and Work to Do

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Sipilä, J.O.T. Adult-Onset Neuroepidemiology in Finland: Lessons to Learn and Work to Do. J. Clin. Med. 2023, 12, 3972. doi:10.3390/jcm12123...
Sunday, June 11, 2023

Jackson Laboratory receives $22.8 million grant for gene-editing based work

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Published: Jun. 10, 2023​. BAR HARBOR, Maine (WABI) - Researchers aiming to develop and validate new gene-editing based therapeutic approach...
Friday, June 9, 2023

Pharmacists are initiators in palliative care for patients with rare diseases

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Dooms, M. Pharmacists are initiators in palliative care for patients with rare diseases. Orphanet J Rare Dis 18, 141 (2023). doi:10.1186/s13...

Targeting Ion Channels and Purkinje Neuron Intrinsic Membrane Excitability as a Therapeutic Strategy for Cerebellar Ataxia

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Huang H, Shakkottai VG. Targeting Ion Channels and Purkinje Neuron Intrinsic Membrane Excitability as a Therapeutic Strategy for Cerebellar ...
Thursday, June 8, 2023

Randomized controlled trial data for successful new drug application for rare diseases in the United States

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Kubota, Y., Narukawa, M. Randomized controlled trial data for successful new drug application for rare diseases in the United States. Orphan...

Ataxia de Friedreich, revisión y actualización de la literatura con búsqueda sistemática de casos en Latinoamérica

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Alfaro-Olivera M, Calle-Nuñez A, Uribe-León A, Aguirre-Quispe W, Sarapura-Castro E, Cornejo-Olivas M. Ataxia de Friedreich, revisión y actua...