Monday, July 14, 2025

RWD132 Onset of Cardiomyopathy and Cardiovascular Disease-Related Burden in Friedreich Ataxia: Real-World Data From Medical Claims

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Sheng-Han Kuo, Boyang Bian, Sarah M. England, Daniel R.J. Gomes, Jim McKay, Tony Wang, Robin L. Avila, Susan Perlman, RWD132 Onset of Cardio...
Sunday, July 13, 2025

False Beliefs, True Deficits: Investigating Social Cognition in Friedreich Ataxia

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Heleven E, Vyhnalek M, Karamazovová S, Van Overwalle F, Naeije G. False Beliefs, True Deficits: Investigating Social Cognition in Friedreich...

Unveiling the Idiographic Portrait of Friedreich's Ataxia in an Omani Patient: A Multidisciplinary Case Stud

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Al-Adawi, Samir and Al Busaidi, Saoud Jaber and Al Jahwari, Nasra Ali and Rajeev, Neeraja and Alriyami, Maha and ALBusaidi, Alya and Al Kind...

Larimar Therapeutics Publishes Nonclinical Data Supporting the Therapeutic Potential of Nomlabofusp in Patients with Friedreich’s Ataxia

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BALA CYNWYD, Pa., July 08, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology comp...

LEXEO THERAPEUTICS ANNOUNCES FDA BREAKTHROUGH THERAPY DESIGNATION FOR LX2006 IN FRIEDREICH ATAXIA

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

Lexeo Therapeutics Announces FDA Breakthrough Therapy Designation for LX2006 in Friedreich Ataxia

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07/07/2025 ;Lexeo Therapeutics (Nasdaq: LXEO) has achieved a significant milestone as its drug candidate LX2006 received FDA Breakthrough Th...
Thursday, July 3, 2025

Antisense oligonucleotide therapy for Friedreich’s ataxia patients carrying the c.165+5G>C splicing mutation

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Antisense oligonucleotide therapy for Friedreich’s ataxia patients carrying the c.165+5G>C splicing mutation. Yameogo, Pouiré et al. Mole...
Friday, June 27, 2025

Biogen Launches Phase 3 Pediatric Trial Of Omaveloxolone For Friedreich Ataxia

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Biogen Inc.June 23, 2025. It has begun dosing in the global Phase 3 BRAVE trial to test omaveloxolone (SKYCLARYS®) in children aged 2 to 16...

The clinical burden of Friedreich ataxia in the United States: A retrospective claims database analysis

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The clinical burden of Friedreich ataxia in the United States: A retrospective claims database analysis, Perlman, Susan et al.. Journal of t...

Stable Isotope Labeling in Bacteria Enables Characterization and Quantification of Frataxin Protein in a Friedreich’s Ataxia Zebrafish Model

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Stable Isotope Labeling in Bacteria Enables Characterization and Quantification of Frataxin Protein in a Friedreich’s Ataxia Zebrafish Model...