Tuesday, July 30, 2024

Microfluidic formulation of diazoxide-loaded solid lipid nanoparticles as a Novel approach for Friedreich's ataxia treatment

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Ilaria Arduino, Antonella Santoro, Silvia De Santis, Rosa Maria Iacobazzi, Angela Assunta Lopedota, Eleonora Paradies, Giuseppe Merla, Sara ...

Phase 2 Trial of Dimethyl Fumarate for Friedrich Ataxia

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NeurologyLive. July 17, 2024. Updates in Therapeutic Development: Clinical Trial Readouts to Watch in the Second Half of 2024  According to ...

Lexeo Therapeutics Announces Positive Interim Phase 1/2 Clinical Data of LX2006 for the Treatment of Friedreich Ataxia Cardiomyopathy

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NEW YORK, July 15, 2024 (GLOBE NEWSWIRE) -- Lexeo Therapeutics, Inc. (Nasdaq: LXEO),  announced positive interim data of LX2006 for the trea...

Anatomical and functional analysis of the corticospinal tract in an FRDA mouse model

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Anatomical and functional analysis of the corticospinal tract in an FRDA mouse model. Misa Nishiyama, John Kalambogias, Fumiyasu Imai, Emily...

Challenges for gene therapy in the financial sustainability of health systems: a scoping review

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Ossandon, H., Armijo, N., Vargas, C. et al. Challenges for gene therapy in the financial sustainability of health systems: a scoping review....

SYSTEMIC FRATAXIN DEFICIENCY CAUSES TISSUE-DEPENDENT IRON HOMEOSTASIS ALTERATIONS: IMPLICATIONS FOR FRIEDREICH ATAXIA

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SYSTEMIC FRATAXIN DEFICIENCY CAUSES TISSUE-DEPENDENT IRON HOMEOSTASIS ALTERATIONS: IMPLICATIONS FOR FRIEDREICH ATAXIA Maria Pazos-Gil, Marta...

Altered calcium responses and antioxidant properties in Friedreich’s ataxia-like cerebellar astrocytes

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Altered calcium responses and antioxidant properties in Friedreich’s ataxia-like cerebellar astrocytes Chiara Marullo, Laura Croci, Iris Giu...

Relative Bioavailability of Omaveloxolone When Capsules Are Sprinkled Over and Mixed in Applesauce Compared With Administration as Intact Omaveloxolone Capsules: A Phase 1, Randomized, Open-Label, Single-Dose, Crossover Study in Healthy Adults

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Hynes SM, Goldsberry A, Henneghan PD, et al. Relative bioavailability of omaveloxolone when capsules are sprinkled over and mixed in applesa...
Monday, July 1, 2024

Advancing Understanding of Predictive Factors for Survival in Friedreich's Ataxia: A Review of Current Evidence and Future Directions

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Xia F, Su C. Advancing Understanding of Predictive Factors for Survival in Friedreich's Ataxia: A Review of Current Evidence and Future ...
Thursday, June 20, 2024

C-Path partners with FARA to fortify RDCA-DAP and further accelerate drug development with new Friedreich’s Ataxia Data

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TUCSON, Ariz., June 18, 2024 — Critical Path Institute (C-Path), a leader in accelerating drug development for rare diseases, today announce...