Friday, May 27, 2022

Lexeo to start phase I/II clinical trial for patients with Friedreich’s ataxia cardiomyopathy

›
May 26, 2022 -- Gene therapy company Lexeo Therapeutics in 2022 plans to launch a phase I/II clinical trial of its adeno-associated virus-ba...

Reata Pharmaceuticals Announces FDA Filing Acceptance and Priority Review Designation for the NDA for Omaveloxolone for the Treatment of Patients with Friedreich’s Ataxia

›
May 26, 2022. PLANO, Texas--(BUSINESS WIRE)-Today announced that the U.S. Food and Drug Administration (“FDA”) has accepted for filing and g...
Monday, May 23, 2022

A comprehensive Triple Repeat Primed PCR (TR-PCR) and a Long-Range PCR (LR-PCR) agarose-based assay for improved genotyping of GAA repeats in Friedreich’s Ataxia

›
Mohamed Jama, Rebecca L. Margraf, Ping Yu, N. Scott Reading, Pinar Bayrak-Toydemir; The Journal of Molecular Diagnostics, 2022, doi:10.1016/...
Friday, May 20, 2022

A Drug Combination Rescues Frataxin-Dependent Neural and Cardiac Pathophysiology in FA Models

›
Abeti R, Jasoliya M, Al-Mahdawi S, Pook M, Gonzalez-Robles C, Hui CK, Cortopassi G and Giunti P (2022). Front. Mol. Biosci. 9:830650. doi: 1...
Thursday, May 19, 2022

Towards a metabolomic approach to investigate iron-sulfur cluster biogenesis

›
Marengo M, Fissore A, Oliaro-Bosso S, Adinolfi S, Pastore A.; IUBMB Life. 2022 Apr 27. doi: 10.1002/iub.2618. Epub ahead of print. PMID: 354...

Novel Regulatory Role of SIRT3 on Cardiomyocyte Mitochondrial Frataxin and Ferroptosis

›
Cantrell A, Su H, Zeng H, Chen JX.; FASEB J. 2022 May;36 Suppl 1. doi: 10.1096/fasebj.2022.36.S1.R2008. PMID: 35557151.  We have demonstrat...
Wednesday, May 18, 2022

Reata plan to submit an MAA to the EMA in the fourth quarter of 2022.

›
PLANO, Texas--(BUSINESS WIRE)-- Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” “our,” “us,” or “we”), a clinical-stage ...
Monday, May 16, 2022

Antioxidantes en el tratamiento de la ataxia de Friedreich. Ensayo in vitro de la eficacia del aceite esencial de clavo

›
García Vivancos, Marta; Repositorio Institucional Universitat de les Illes Balears, URI: http://hdl.handle.net/11201/157022, Fecha: 2021, Fe...
Sunday, May 15, 2022

EU/3/21/2456: Orphan designation for the treatment of Friedreich's ataxia. Withdrawn

›
Sponsor: Novartis Gene Therapies EU Limited The designation was withdrawn from the Union Register of orphan medicinal products in March 202...

Neurological Recovery with Interferon-gamma Treatment in Friedreich's Ataxia

›
Tekin HG, Levent E.; J Coll Physicians Surg Pak. 2022 May;32(5):671-673. doi: 10.29271/jcpsp.2022.05.671. PMID: 35546709.  The treatment was...