Wednesday, October 29, 2025

Drivers of managed entry agreements to reduce reimbursement challenges of orphan medicinal products: the development of a matrix

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Callenbach, M.H.E., van den Berg, S., Hulsbosch, A. et al. Drivers of managed entry agreements to reduce reimbursement challenges of orphan ...
Saturday, October 25, 2025

Positron emission tomography reveals increased myocardial glucose uptake in a subset of Friedreich ataxia patients

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Payne, R.M., O’Connell, T.M., Pride, P.M. et al. Positron emission tomography reveals increased myocardial glucose uptake in a subset of Fri...

Development of an AAV-Based Gene Therapy for the Ocular Phenotype of Friedreich’s Ataxia

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Tang H, Gupte S, Xu E, Calabro KR, Friend H, Crosson SM, Fajardo D,Kostamo Z, Zhang H, Peterson JJ, Lin F, Kozmik Z, Lutz CM, Boye SL, Boye ...
Friday, October 24, 2025

Neuropsychiatric challenges of Friedreich ataxia in a patient residing in a long-term care facility

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Wong J, Kwok J, Kim K. Neuropsychiatric challenges of Friedreich ataxia in a patient residing in a long-term care facility. Prim Care Compan...
Thursday, October 23, 2025

Jupiter Neurosciences, Inc. strategic partnership with Zina Biopharmaceutica

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October 22, 2025. The company develops JNS101, which is in Phase II trial for the treatment of Friedreich's Ataxia, a rare inherited dis...
Wednesday, October 22, 2025

Exploring the pleiotropic effects of lncRNA in different repeat expansion disorders

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Soumalya Das, Shubhi Khandelwal, Sakshi Shukla, Amit Kumar, Exploring the pleiotropic effects of lncRNA in different repeat expansion disord...
Monday, October 20, 2025

A Probabilistic Deep Ensemble Framework for the Objective Assessment of Friedreich Ataxia

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Maneesha Randeniya, Kanishka Ranaweera, Thang Ngo, et al. A Probabilistic Deep Ensemble Framework for the Objective Assessment of Friedreich...

Chapter 23 - Omaveloxolone: a nuclear factor erythroid 2-related factor 2 activator for Friedreich’s ataxia

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Ziquan Zhao, Junjie Wang, Mengchen Lu, Qidong You, Zhengyu Jiang, Chapter 23 - Omaveloxolone: a nuclear factor erythroid 2-related factor 2 ...
Thursday, October 16, 2025

Targeting rare splicing defects: Antisense oligonucleotides offer a therapeutic strategy in FRDA

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Targeting rare splicing defects: Antisense oligonucleotides offer a therapeutic strategy in FRDA, Kerkhof, Laurie M.C. et al., Molecular The...
Monday, October 13, 2025

Analysis of a Modified Version of the Inventory of Non-Ataxia Signs Over 12 Years in Patients with Friedreich's Ataxia in the EFACTS Study

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Lischewski, S.A., Dogan, I., Giunti, P., Parkinson, M.H., Mariotti, C., Durr, A., Ewenczyk, C., Boesch, S., Nachbauer, W., Klopstock, T., St...