Friday, January 30, 2026

Larimar’s Pediatric Friedreich’s Ataxia Trial Termination: What Investors Should Know

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TipRanks Clinical-Trials-Jan 28, 2026  "The study was first submitted in November 2024, signaling the formal start of the regulatory pr...
Wednesday, January 14, 2026

California grants $7.4 million to advance gene-edited stem cell therapy for Friedreich’ s ataxia

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Gen 13, 2026. The California Institute for Regenerative Medicine (CIRM) has awarded $7.4 million to support a University of California San D...

Orphan Drug Designation for SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

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CHARLESTOWN, Mass., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. today announced that U.S. Food and Drug Administration (FDA) ha...
Monday, January 12, 2026

Participant in First-in-Class Phase 1b FALCON Trial Evaluating SGT-212 Dual-Route Gene Therapy for the Treatment of Friedreich’s Ataxia

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CHARLESTOWN, Mass., Jan. 12, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. today announced that the first participant has been dosed in FA...
Saturday, January 10, 2026

Biogen Advances New SKYCLARYS Formulation in Early Trial, Supporting Long-Term Rare Disease Strategy

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TipRanks Clinical-Trials-Auto-Generated Newsdesk, Jan 09, 2026. Study Overview This Phase 1 Biogen study, officially titled “A Phase 1, Rand...
Friday, January 9, 2026

Insights into DNA repeat expansions among 900,000 biobank participants

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Hujoel, M.L.A., Handsaker, R.E., Tang, D. et al. Insights into DNA repeat expansions among 900,000 biobank participants. Nature (2026). doi:...
Thursday, January 8, 2026

Pathological frataxin deficiency in mice causes tissue-specific alterations in iron homeostasis

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Pazos-Gil, M., Medina-Carbonero, M., Sanz-Alcázar, A., Portillo-Carrasquer,M., Oliveira-Jorge, L., Hernández, G., Sánchez, M., Delaspre, F.,...
Wednesday, January 7, 2026

Microgliopathy as a primary mediator of neuronal death in models of Friedreich's Ataxia

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Pernaci C, Johnson A, Gillette S, Warden AS, McCormick C, Weiser-Novak S, Ramirez G, Broersma EH, Mishra P, Sivakumar A, Cherqui S, Coufal N...
Tuesday, January 6, 2026

Nanobodies as tools for studying human frataxin biology

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Pignataro, M.F., Fernández, N.B., Garay-Alvarez, A. et al. Nanobodies as tools for studying human frataxin biology. Commun Biol (2026). doi:...

Ataxia with hypertonia and absent deep tendon reflexes (DTRs)

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Maddela, Chakradhar. (2026). Ataxia with hypertonia and absent deep tendon reflexes (DTRs) -DD. 10.13140/RG.2.2.12440.66562.  It is an impor...