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, ...
Wednesday, March 6, 2024

Vestibular Pathology as Early Finding of Friedreich's Ataxia in a 16 Years Old Woman

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Fernández AG. Vestibular Pathology as Early Finding of Friedreich's Ataxia in a 16 Years Old Woman. Indian J Otolaryngol Head Neck Surg....
Saturday, March 2, 2024

Frataxin Traps Low Abundance Quaternary Structure to Stimulate Human Fe-S Cluster Biosynthesis

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1. Cory S, Lin C-W, Havens S, Patra S, Putnam C, Shirzadeh M, et al. Frataxin Traps Low Abundance Quaternary Structure to Stimulate Human Fe...

Characterization of Cardiac-Onset Initial Presentation in Friedreich Ataxia

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Lynch, D.R., Subramony, S., Lin, K.Y. et al. Characterization of Cardiac-Onset Initial Presentation in Friedreich Ataxia. Pediatr Cardiol (2...
Friday, March 1, 2024

Prime Medicine Reports Full Year 2023 Financial Results and Provides Business Updat

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CAMBRIDGE, Mass., March 01, 2024 (GLOBE NEWSWIRE) -- Prime Medicine, Inc. (Nasdaq: PRME), a biotechnology company committed to delivering a ...

After pair of failures, PTC finds a path through FDA jungle for Friedreich ataxia med

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https://www.fiercebiotech.com. By Annalee Armstrong; Mar 1, 2024. In a fourth-quarter earnings update Thursday afternoon, PTC executives bri...

PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2023 Financial Results

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SOUTH PLAINFIELD, N.J., Feb. 29, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. PTC had a Type C meeting with the FDA in the first quarter of 2...
Monday, February 26, 2024

Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich’s Ataxia in Collaboration with Neurocrine Biosciences

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Voyager Therapeutics Announces Selection of Gene Therapy Development Candidate for Friedreich’s Ataxia in Collaboration with Neurocrine Bios...
Sunday, February 25, 2024

Phenotypic variation of FXN compound heterozygotes in a Friedreich ataxia cohort

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Shen MM, Rummey C, Lynch DR. Phenotypic variation of FXN compound heterozygotes in a Friedreich ataxia cohort. Ann Clin Transl Neurol. 2024 ...