Saturday, May 11, 2024

1. WO2024097772 - COMPOSITIONS AND METHODS FOR TREATMENT OF FRIEDREICH'S ATAXIA

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N.º de publicación WO/2024/097772. Fecha de publicación 10.05.2024. Nº de la solicitud internacional PCT/US2023/078373. Fecha de presentació...
Friday, May 10, 2024

Ferrostatin-1 specifically targets mitochondrial iron-sulfur clusters and aconitase to improve cardiac function in Sirtuin 3 cardiomyocyte knockout mice

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Ferrostatin-1 specifically targets mitochondrial iron-sulfur clusters and aconitase to improve cardiac function in Sirtuin 3 cardiomyocyte k...

Larimar Therapeutics Reports First Quarter 2024 Operating and Financial Results

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BALA CYNWYD, Pa., May 09, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. In March 2024, the first patient was dosed in the OLE study ev...
Thursday, May 9, 2024

Stealth BioTherapeutics’ SBT-589 Shows Cardioprotective Effects in Preclinical Models of Friedreich Ataxia

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May 3, 2024. New data on SBT-589 (Stealth BioTherapeutics) presented at the 2024 Wellcome Trust Conference on Mitochondrial Medicine – Thera...

Design Therapeutics Announces First Quarter 2024 Financial Results and Highlights Upcoming Program Milestones

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CARLSBAD, Calif., May 08, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc.Friedreich Ataxia (FA) Design’s new drug product for FA, DT-216P...
Friday, May 3, 2024

Elevated Bile Acid 3β,5α,6β-Trihydroxycholanoyl Glycine in a Subset of Adult Ataxias Including Niemann–Pick Type C

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Motamed-Gorji, N.; Khalil, Y.; Gonzalez-Robles, C.; Khan, S.; Mills, P.; Garcia-Moreno, H.; Ging, H.; Tariq, A.; Clayton, P.T.; Giunti, P. E...
Thursday, May 2, 2024

Calcitriol Treatment Is Safe and Increases Frataxin Levels in Friedreich Ataxia Patients

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Alemany-Perna, B., Tamarit, J., Cabiscol, E., Delaspre, F., Miguela, A., Huertas-Pons, J.M., Quiroga-Varela, A., Merchan Ruiz, M., López Dom...

Genetic Determined Iron Starvation Signature in Friedreich's Ataxia

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Grander, M., Haschka, D., Indelicato, E., Kremser, C., Amprosi, M., Nachbauer, W., Henninger, B., Stefani, A., Högl, B., Fischer, C., Seifer...

One-to-one Benefit provided by Antioxidants to cultured skin Fibroblasts from Friedreich Ataxia patients

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One-to-one Benefit provided by Antioxidants to cultured skin Fibroblasts from Friedreich Ataxia patients, Paule Bénit, Malgorzata Rak, Pierr...

Astellas' Friedreich’s ataxia gene therapy cleared for clinical study after earlier version stumbled

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April 25, 2024. Astellas' Friedreich’s ataxia gene therapy cleared for clinical study after earlier version stumbled. On Thursday, the J...