Wednesday, March 20, 2024

Stealth BioTherapeutics Presents Data of Novel Compound, SBT-589, in Friedreich's Ataxia Cardiac Models at the Wellcome Trust Mitochondrial Medicine Conference

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NEEDHAM, Mass., March 19, 2024 /PRNewswire/ -- Stealth Biotherapeutics Inc.  SBT-589 showed mitochondrial protection across models of Friedr...
Tuesday, March 19, 2024

Design Therapeutics Outlines Progress Across GeneTAC™ Platform and Announces Fourth Quarter and Full Year 2023 Financial Results

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CARLSBAD, Calif., March 19, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc..  New Drug Product for Friedreich Ataxia (FA) DT-216P2 with F...

Accelerometer-based measures in Friedreich ataxia: a longitudinal study on real-life activity

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Accelerometer-based measures in Friedreich ataxia: a longitudinal study on real-life activity. Front. Pharmacol., 19 March 2024, Sec. Neurop...
Monday, March 18, 2024

P003: Clinical laboratory experience of frataxin quantification in blood for the diagnosis of Friedreich ataxia*

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Iris Pantovich, Amy White, April Studinski, Weiyi Mu, Bonnie Kaas, Matthew Bower, Gisele Pino, Dawn Peck, Kyle Salsbery, Emily Lauer, Angela...
Thursday, March 14, 2024

Larimar Therapeutics Reports Fourth Quarter and Full Year 2023 Operating and Financial Results and Provides Update on Nomlabofusp Development

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BALA CYNWYD, Pa., March 14, 2024 (GLOBE NEWSWIRE).  Positive top-line data from Phase 2 dose exploration study of nomlabofusp, which was gen...

Standing Balance Conditions and Digital Sway Measures for Clinical Trials of Friedreich's Ataxia

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Casey, H.L., Shah, V.V., Muzyka, D., McNames, J., El-Gohary, M., Sowalsky, K., Safarpour, D., Carlson-Kuhta, P., Schmahmann, J.D., Rosenthal...

Larimar Therapeutics Announces the Dosing of the First Patient in Long-term Open Label Extension Study for Nomlabofusp in Patients with Friedreich’s Ataxia

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BALA CYNWYD, Pa., March 11, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology c...

Lexeo Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Operational Highlights

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NEW YORK, March 11, 2024 (GLOBE NEWSWIRE). LX2006 for the Treatment of FA Cardiomyopathy: Announced preliminary frataxin protein expression ...
Wednesday, March 13, 2024

Generation and characterization of iPSC lines from Friedreich’s ataxia patient (FRDA) with GAA.TTC repeat expansion in the Frataxin (FXN) gene’s first intron (IGIBi016-A) and a non-FRDA healthy control individual (IGIBi017-A)

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Istaq Ahmad, Asangla Kamai, Sana Zahra, Himanshi Kapoor, Achal Kumar Srivastava, Mohammed Faruq, Generation and characterization of iPSC lin...

Regulatory Pathway Widening for Cardiac Gene Therapy Hopefuls

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Published: Mar 11, 2024. www.biospace.com  Lexeo Therapeutics is developing a gene therapy, LX2006, for Friedreich’s ataxia cardiomyopathy, ...