Monday, August 3, 2026

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

›
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

›
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

›
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

›
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

›
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

›
Yang W, Thompson B, Miellet S, Maddock M, Napierala M, Dottori M, Kwa FAA. Unlocking Sulforaphane's Potential in Friedreich Ataxia: Furt...
Tuesday, July 21, 2026

Cellares and Papillon Therapeutics Partner to Automate Manufacturing of PPL-001 for Patients With Friedreich’s Ataxia

›
SOUTH SAN FRANCISCO, Calif. & SAN DIEGO, Jul 21, 2026. Cellares, the first Integrated Development and Manufacturing Organization (IDMO),...
Thursday, July 16, 2026

Unveiling the idiographic portrait of Friedreich’s ataxia in an Omani patient: A multidisciplinary case study

›
Al Azri, F. H., Otaify, G. A., AlRiyami, M., Gujjar, A. R., ALBusaidi, A., Al Kindi, F. A., Rajeev, N., Al Busaidi, S. J., Bolourkesh, H., A...
Monday, July 13, 2026

Precision therapeutics and innovative clinical trial design in neurodegenerative diseases

›
Ariadna Domínguez-García, Juan Carlos Delgado-Uriarte, Amin Cervantes-Arriaga, Precision therapeutics and innovative clinical trial design i...

Mitochondria setting the stage for ferroptosis

›
Ahola S. Mitochondria setting the stage for ferroptosis. Trends in Endocrinology & Metabolism, 2026; 0. Doi:10.1016/j.tem.2026.06.006  F...