Saturday, January 10, 2026

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

›
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

›
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

›
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

›
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

›
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)

›
Maddela, Chakradhar. (2026). Ataxia with hypertonia and absent deep tendon reflexes (DTRs) -DD. 10.13140/RG.2.2.12440.66562.  It is an impor...
Monday, January 5, 2026

Patient-reported, psychosocial and health economic outcomes in mild to moderate Friedreich's ataxia: baseline results of the PROFA study

›
Grobe-Einsler M, Borel S, Buchholz M, Sayah S, Hilab R, Pierron L, Iskandar A, Humphries B, Ewenczyk C, Heinzmann A, Atencio M, Feldmann K, ...
Tuesday, December 30, 2025

Development of a secretable frataxin for enhanced efficacy in treating Friedreich’s Ataxia

›
Daniel M. DuBreuil, Michael Fleming, Yashvi Parikh, Mikaela Woo, Jie Bu, Swathi Ayloo, Ingeborg M. Langohr, Dinesh S. Bangari, Christian Mue...

Using a discrete choice experiments to explore societal preferences for valuing new drugs for rare diseases

›
Vargas, C., Goodall, S., Street, D.J. et al. Using a discrete choice experiments to explore societal preferences for valuing new drugs for r...
Monday, December 29, 2025

Progressive scoliosis in a pediatric patient with cerebellar ataxia: surgical challenges and literature review

›
Asunis E, Vitulli F, Nicotra R, Rubino A, Cicala D, Cinalli G, Colella G. Progressive scoliosis in a pediatric patient with cerebellar ataxi...