Monday, October 17, 2022

Selection of Synthetic Proteins to Modulate the Human Frataxin Function

›
Pignataro MF, Herrera MG, Fernández N, Aran M, Gentili H, Bataglini F, Santos J.; Biotechnol Bioeng. 2022 Oct 12. doi: 10.1002/bit.28263. Ep...
Friday, October 14, 2022

Reata Pharmaceuticals Announces that the FDA Does Not Plan to Hold an Advisory Committee Meeting to Discuss the NDA for Omaveloxolone for Friedreich’s Ataxia

›
October 13, 2022. PLANO, Texas--(BUSINESS WIRE)-- Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” “our,” “us,” or “we”),...
Thursday, October 13, 2022

Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia

›
ClinicalTrials.gov Identifier: NCT05573698. A Phase 1b, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose and Multi-Dose...
Wednesday, October 12, 2022

SARS-CoV-2 in patients with Friedreich ataxia

›
Megan M. Shen, Layne N. Rodden, Kellie McIntyre, Adriana Arias, Victoria Profeta, Kimberly Schadt & David R. Lynch; J Neurol (2022). do...

A promising mouse model for Friedreich Ataxia progressing like human patients

›
Catherine Gérard, Annabelle Fortin Archambault, Camille Bouchard, Jacques P. Tremblay; Behavioural Brain Research, Volume 436, 2023, 114107,...
Tuesday, October 4, 2022

Diabetes mellitus y ataxia de Friedreich en un niño: una difícil coexistencia [Diabetes mellitus and Friedreich´s ataxia in a child: a complicated coexistence]

›
Marqués Cabrero A, Expósito Raspeño M, Sánchez Escudero V, Gutiérrez Cruz N, González Vergaz A.; Arch Argent Pediatr. 2022 Oct;120(5):e223-e...
Monday, October 3, 2022

Plasma multi-omics analysis reveals very long chain ceramides as validated biomarkers of Friedreich’s ataxia

›
Dezhen Wang, M. Grazia Cotticelli, Blanca E. Himes, David R. Lynch, Clementina Mesaros; medRxiv 2022.09.27.22280432; doi:10.1101/2022.09.27....

Solid Biosciences Announces Acquisition of AavantiBio and Concurrent $75 Million Private Placement

›
September 30, 2022 07:00 ET | Source: Solid Biosciences Inc CHARLESTOWN, Mass. and CAMBRIDGE, Mass., Sept. 30, 2022 (GLOBE NEWSWIRE) -- Sol...

Friedreich Ataxia: An expanded access program for Elamipretide treatment

›
Children's Mercy Kansas City / Children’s Mercy Research Institute.  Full Study Name: SPIES-006 This study is for participants who ...
Wednesday, September 28, 2022

Guía de Evaluación Diagnóstica y Discapacidad en Pacientes con Ataxias y Paraparesias Espásticas Hereditarias

›
Francisco Javier Arpa Gutiérrez, María José Abenza Abildúa, Idoia Rouco Axpe. Sociedad Española de Neurología (2022). ISBN: 978-84-124320-4-...