Tuesday, August 4, 2026

Larimar Therapeutics Reports Positive Open Label Data and Submission of First Module of Rolling BLA for Accelerated Approval of Nomlabofusp for Friedreich’s Ataxia

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June 29, 2026. BALA CYNWYD, Pa., June 29, 2026 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar)  a clinical-stage biotechnology comp...

Characterizing Friedreich’s ataxia cardiomyopathy with serial cardiac magnetic resonance imagings

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Cripe, L. H., Alvarado, C., Hayes, E. A., Krishnamurthy, R., Gunsaulus, M. E., Waldrop, M. A., & Nandi, D. (2026). Characterizing Friedr...

Effects of Friedeich Ataxia on the Interrelations Between Left Ventricular Length and Long-Axis Systolic and Diastolic Excursions

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R. Peverill, Effects of Friedeich Ataxia on the Interrelations Between Left Ventricular Length and Long-Axis Systolic and Diastolic Excursio...

Cardiomyocyte Dysfunction is Modulated by PCDHGA10 in Friedreich Ataxia,

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J. Lees, H. Zhang, L. Jiao, A. Kong, R. Phang, L. Li, N. Su, A. Mukhtar, S. Bass-Stringer, A. Pébay, M. Dottori, L. Corben, M. Delatycki, R....
Monday, August 3, 2026

Design Therapeutics Provides RESTORE-FA Clinical Development Update and Reports Second Quarter 2026 Financial Results

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CARLSBAD, Calif., Aug. 03, 2026 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc..Positive  RESTORE-FA Four-Week Data Support Advancement of DT-...

Towards routine genetic testing of repeat expansions in neurogenetic diseases using multiplex CRISPR-Cas9-targeted long read sequencing

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Fergelot, P., Boury, C., Penaud, B. et al. Towards routine genetic testing of repeat expansions in neurogenetic diseases using multiplex CRI...

Clinical Challenges in Managing Diabetes Mellitus in Friedreich’s Ataxia

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Aarya Naik, MBBS, Hooman Oktaei, MD, Clinical Challenges in Managing Diabetes Mellitus in Friedreich’s Ataxia, Endocrine Practice , 32, S66-...
Saturday, July 25, 2026

CRISPR-Cas9-based therapies for Huntington's disease and Friedreich's ataxia: mechanisms, advances, and future perspectives

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Mundada AR, Badikol AR, Mangu K. CRISPR-Cas9-based therapies for Huntington's disease and Friedreich's ataxia: mechanisms, advances,...
Thursday, July 23, 2026

Microglia from Friedreich Ataxia patients are intrinsically primed for neuroinflammation

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La microglía de Friedreich Ataxia de los pacientes está intrínsecamente preparada para la neuroinflamación Ye Man Tang, Rita Lo, Louise Thir...
Wednesday, July 22, 2026

Unlocking Sulforaphane's Potential in Friedreich Ataxia: Further Evidence from Preclinical Investigations Using Induced Pluripotent Stem Cell-Derived Sensory Neurons

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Yang W, Thompson B, Miellet S, Maddock M, Napierala M, Dottori M, Kwa FAA. Unlocking Sulforaphane's Potential in Friedreich Ataxia: Furt...