Monday, August 18, 2025

Propensity-matched analysis comparing omaveloxolone treatment to Friedreich ataxia natural history data: a plain language summary

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Lynch, D. R., Goldsberry, A., Rummey, C., Farmer, J., Boesch, S., Delatycki, M. B., … Meyer, C. (2025). Propensity-matched analysis comparin...

Sensory nerve action potential reappearance after omaveloxolone treatment in patients with Friedreich ataxia

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Sensory nerve action potential reappearance after omaveloxolone treatment in patients with Friedreich ataxia. Melita Rotar, Lea Leonardis; C...

Best Oculomotor Endpoints for Clinical Trials in Hereditary Ataxias: A Systematic Review and Consensus by the Ataxia Global Initiative Working Group on Digital‑Motor Biomarkers

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Pretegiani E, Garces P, Antoniades CA, Sobanska A, Kovacs N, Ying SH, Gupta AS, Perlman S, Szmulewicz DJ, Pane C, Németh AH, Jardim LB, Coar...
Sunday, August 17, 2025

FRIEDREICH ATAXIA- STEROIDOGENESIS (FRIEDSTERO)

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ClinicalTrials.gov ID NCT07123142. Sponsor Istanbul University Information provided by Ozge Bayrak Demirel, Istanbul University (Responsible...
Saturday, August 16, 2025

Larimar Therapeutics Reports Second Quarter 2025 Financial Results

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BALA CYNWYD, Pa., Aug. 14, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar). Initial data from the 50 mg dose in the open label...

Lexeo Therapeutics Reports Second Quarter 2025 Financial Results and Operational Highlights

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NEW YORK, Aug. 14, 2025 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc.​. Breakthrough Therapy designation granted for LX2006 based on interim ...
Thursday, August 14, 2025

Solid Biosciences Inc. Reports Progress in Clinical Trials and Financial Highlights for Q2 2025

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Quiver Quantitative. Aug. 12, 2025 SGT-212 for Friedreich’s Ataxia (FA) The Company expects to initiate a first-in-human, open-label, Phase ...
Saturday, August 9, 2025

Manejo anestésico de tempestade tireoidiana em paciente com ataxia de Friederich. Relato de caso

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Giovanna Calixto Rossi Marques de Souza, Laura Faleiros de Lima, Paula Ariane Toneli Reis. Archives of Health, Curitiba, v.6, n.4, special e...

Clinical and cognitive assessment in Friedreich ataxia clinical trials: a review

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Darriba Á, Munnich A, Cardoso-Leite P, Funalot B, Waszak F. Clinical and cognitive assessment in Friedreich ataxia clinical trials: a review...

Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich's Ataxia

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Conditions: Friedreich Ataxia Interventions: Drug: SKYCLARYS (omaveloxolone) Sponsors: Centre Hospitalier Universitaire de Nice  Not yet rec...