Tuesday, August 4, 2026

Stem Cell Transplantation in Friedreich Ataxia: Cure for Leukemia but No Effect on Neurological Progression

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A. Gitman, N. Bhandari, M. Castellaro, K. Schadt, M. Cancio, and D. R. Lynch, “ Stem Cell Transplantation in Friedreich Ataxia: Cure for Leu...

Adjuvia Therapeutics Closes $8 Million Series Seed Financing to Advance Novel Mitochondrial Disease Therapy into the Clinic

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Jul 29, 2026. SAN FRANCISCO--(BUSINESS WIRE)--Adjuvia Therapeutics, a biotechnology company developing therapeutics to treat mitochondrial d...

Goldenrod Therapeuticsare committed to developing a next-generation phosphodiesterase 4 (PDE4) inhibitor designed to address serious neurological conditions, including Friedreich’s ataxia

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Goldenrod Therapeutics is a portfolio company of Fannin based in Houston, Texas. We are committed to developing a next-generation phosphodie...

Neurophysiological assessment of disease severity in Friedreich’s Ataxia: a study of brainstem auditory and visual evoked potentials

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Maccora S, Quartetti U, Lima SM, Rini N, Cucchiara M, Agnello L, Gambino CM, Brighina F, Ciaccio M, Di Stefano V. Neurophysiological assessm...

Omaveloxolone Driven NRF2 Activation as a Novel Therapeutic Strategy for Pulmonary Hypertension

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Omaveloxolone Driven NRF2 Activation as a Novel Therapeutic Strategy for Pulmonary Hypertension. Huang, Chuangjia, Bai, June, Luo, Ang, Yan...

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-...