Wednesday, April 2, 2025

Larimar Therapeutics Provides Nomlabofusp Development Update and Reports Fourth Quarter and Full Year 2024 Financial Results

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BALA CYNWYD, Pa., March 24, 2025 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) Potential for Accelerated Approval Pathway Based o...

Comparative analysis of large language models on rare disease identification

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Ao, G., Chen, M., Li, J. et al. Comparative analysis of large language models on rare disease identification. Orphanet J Rare Dis 20, 150 (2...

Early onset development of hypertrophic cardiomyopathy in less than 1 year in a patient with familial Friedrich's ataxia: Case report

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Yasmine Ouaddouh, Salma Bouyaddid, Zakaria Bazid, Nabila Ismaili, Noha El Ouafi, Early onset development of hypertrophic cardiomyopathy in l...

Jupiter Neurosciences Inc. (JUNS) reports earnings

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The report was filed on March 28, 2025. JOTROL is being developed for multiple indications including Parkinson’s Disease, Mild Cognitive Imp...
Wednesday, March 26, 2025

Redox homeostasis and Inflammation in fibroblasts of patients with Friedreich Ataxia: a possible cross talk

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Andrea Quatrana, Sara Petrillo Sara Petrillo, Caterina Torda Caterina Torda, Eleonora De Santis, Enrico Bertini Enrico Bertini, Fiorella Pie...

Systemic AAV Gene Therapy with Next Generation Engineered Capsids for Treatment of CNS and Cardiac Symptoms in Friedreich’s Ataxia

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MDA Conference 2025. Ryan Kast, PhD, Capsida Biotherapeutics, Celeste Stephany, PhD, Capsida Biotherapeutics, Miguel Chuapoco, PhD, Capsida ...
Tuesday, March 25, 2025

LEXEO THERAPEUTICS REPORTS FOURTH QUARTER AND FULL YEAR 2024 FINANCIAL RESULTS AND OPERATIONAL HIGHLIGHTS

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NEW YORK, March 24, 2025 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. .  Mid-Year Clinical Update Expected to Include:  Safety and tolerabil...
Monday, March 24, 2025

Neuroimaging Biomarkers for Friedreich Ataxia: A Cross-Sectional Analysis of the TRACK-FA Study

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Georgiou-Karistianis, N., Corben, L.A., Lock, E.F., Bujalka, H., Adanyeguh, I., Corti, M., Deelchand, D.K., Delatycki, M.B., Dogan, I., Farm...
Friday, March 21, 2025

Debates over orphan drug pricing: a meta-narrative literature review

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Hanchard, M.S. Debates over orphan drug pricing: a meta-narrative literature review. Orphanet J Rare Dis 20, 107 (2025). doi:10.1186/s13023-...

Brain microvascular endothelial cells differentiated from a Friedreich's Ataxia patient iPSC are deficient in tight junction protein expression and paracellularly permeable

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Brain microvascular endothelial cells differentiated from a Friedreich's Ataxia patient iPSC are deficient in tight junction protein exp...