Sunday, April 27, 2025

Employment and work ability in individuals living with rare diseases: a systematic literature review

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Bougas, N., Kangas, T., Vanthomme, K. et al. Employment and work ability in individuals living with rare diseases: a systematic literature r...

Biogen are withdrawing from the NICE appraisal for Skyclarys

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Biogen. 24th April 2025​, Following your request for information, I want to inform you that Biogen has withdrawn omaveloxolone from the Nati...

Artificial Intelligence‐Based Virtual Assistant for the Diagnostic Approach of Chronic Ataxias

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Alessandro, L., Bianciotti, N., Salama, L., Volmaro, S., Navarrine, V., Ameghino, L., … & Rossi, M. Artificial Intelligence‐Based Virtua...

Dietary and lifestyle interventions for the management of hereditary ataxias

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Yang Wenyao , Thompson Bruce , Kwa Faith A. A. ;Dietary and lifestyle interventions for the management of hereditary ataxias. Frontiers in N...

MHRA approves first UK treatment for Friedreich’s ataxia, omaveloxolone

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The Medicines and Healthcare products Regulatory Agency (MHRA) has today, 23 April, approved omaveloxolone (Skyclarys), the first treatment ...
Monday, April 7, 2025

Positive Interim Phase 1/2 Data for LX2006 in Friedreich Ataxia Cardiomyopathy Supporting Advancement to Registrational Study

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NEW YORK, April 07, 2025 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO), a clinical stage genetic medicine company dedicated to...

Case Report: Complex Cardiac Arrhythmia Management in the ICU for an Adolescent with Friedreich Ataxia

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Oluwatomini A Fashina, Stephen J Gleich, Derek N Opp, Yves Ouellette, Yu Kawai; Front. Pediatr. Sec. Pediatric Critical Care Volume 13 - 202...
Sunday, April 6, 2025

Dietary and Lifestyle Interventions for the Management of Hereditary Ataxias

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Wenyao Yang, Bruce Thompson, Faith Kwa; Front. Nutr. Sec. Nutrition and Metabolism Volume 12 - 2025, doi: 10.3389/fnut.2025.1548821 Currentl...

Friedreich’s ataxia treatment’s sexual dimorphism

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Ferreira, J. Friedreich’s ataxia treatment’s sexual dimorphism. Lab Anim 54, 75 (2025). doi:10.1038/s41684-025-01535-2  While NRF2 activatio...
Wednesday, April 2, 2025

Larimar Therapeutics Provides Nomlabofusp Development Update and Reports Fourth Quarter and Full Year 2024 Financial Results

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BALA CYNWYD, Pa., March 24, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) Potential for Accelerated Approval Pathway Based o...