Thursday, November 13, 2025

Cracking the code: a head-to-head comparison of expert clinicians and artificial intelligence in diagnosing rare diseases

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Sendtner, G.W., Muecke, M., Grigull, L. et al. Cracking the code: a head-to-head comparison of expert clinicians and artificial intelligence...
Tuesday, November 11, 2025

Deep learning-based 3D reconstruction of dentate nuclei in Friedreich’s ataxia from T2*weighted MR images

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Trushal Sardhara, Ravi Dadsena, Roland C. Aydin, Ralf-Dieter Hilgers, Leon Horn, Jörg B. Schulz, Kathrin Reetz, Sandro Romanzetti, Imis Doga...
Monday, November 10, 2025

Voyager Reports Third Quarter 2025 Financial and Operating Results

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LEXINGTON, Mass., Nov. 10, 2025 (GLOBE NEWSWIRE) -- Voyager Therapeutics, Inc. (Nasdaq: VYGR), a biotechnology company dedicated to leveragi...

Larimar Therapeutics, Inc. Updates on Nomlabofusp Development

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On November 10, 2025, Larimar Therapeutics, Inc. provided updates on its nomlabofusp clinical program and future plans.
Saturday, November 8, 2025

Individualized exercise and NAD+ precursor supplementation in Friedreich’s Ataxia: a randomized controlled trial

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Lin, Kimberly Y. and Lin, Kimberly Y. and Bucha, Anna and Bucha, Anna and McSweeney, Kara and McSweeney, Kara and Wade, Kristin L. and Wade,...
Wednesday, November 5, 2025

The Triple Flexion Response in Friedrich’s Ataxia

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Saluja A, Sahib A, Yadav V (November 04, 2025) The Triple Flexion Response in Friedrich’s Ataxia. Cureus 17(11): e96080. doi:10.7759/cureus....
Monday, November 3, 2025

Solid Biosciences Reports Third Quarter 2025 Financial Results

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CHARLESTOWN, MA, Nov. 03, 2025 FA (SGT-212): Solid has activated the first clinical trial site and is currently screening participants for ...

Early experience on omaveloxolone in adult patients with Friedreich's ataxia: a real-world observational study

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Lima SM, Caltagirone M, Messina C, Quartetti U, Rini N, D'Amico F, Brighina F, Di Stefano V. Early experience on omaveloxolone in adult ...
Sunday, November 2, 2025

Friedreich ataxia:

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Subramony SH, Lynch DR. Friedreich Ataxia. Pediatr Neurol. 2025 Nov 1;174:148-154. doi: 10.1016/j.pediatrneurol.2025.10.020. Epub ahead of p...
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 ...