Gene editing of human hematopoietic stem and progenitor cells for Friedreich’s ataxia using CRISPR/Cas9 technology Dr. Celine Rocca 1, Ms. Shi Yanmeng 1, Mr. Jay Sharma 1, Dr. Prashant Mali 1, Dr. Stephanie Cherqui 1 University of California, San Diego Our goal is now to develop an autologous HSPC...
Friedreich Ataxia Collaborative Clinical Research Network and Natural History Research Project We want to inform you that Dr. Antoine Duquette, a neurologist at the CHUM, and his research team will now be responsible for a site in Montreal that integrates the Collaborative Research Network in Friedreich Ataxia (CCRN in FA)...
ACHIEVED PRIMARY ENDPOINT OF STATISTICALLY SIGNIFICANT IMPROVEMENT IN MFARS COMPARED TO PLACEBO AFTER 48 WEEKS OF TREATMENT Reata Pharmaceuticals, Inc. (Nasdaq: RETA), a clinical-stage biopharmaceutical company, announced today that the registrational Part 2 portion of the MOXIe Phase 2 trial of omaveloxolone in patients with Friedreich’s ataxia (FA) met its...
Mataró, Barcelona, Spain and Charleroi, Belgium, October 8, 2019 – Minoryx Therapeutics, a company specializing in the development of innovative treatments for orphan central nervous system (CNS) diseases, today announces that it has completed recruitment in the FRAMES phase 2 clinical trial of its novel PPARγ agonist, leriglitazone (MIN-102), in patients...
This is a review article focusing on the insights into the pathogenesis of this disorder and how those insights are being translated into novel therapeutic approaches. For example, more recently, a role for inflammation has emerged as being important in the pathogenesis of Friedreich ataxia. These findings have led to...
The aim of the present study was to characterize and analyze the most important individual and organizational variables associated with job accommodation in subjects with degenerative cerebellar ataxia by administering a series of international and validated work activity-related scales. Twenty-four workers (W) and 58 non-workers (NW) were recruited: 34 with...
For this tenth edition that is Ataxia and I, Manu will talk to you about the importance of friendship for her – for most of the handicapped probably – but also of her need to have often her moments of loneliness! Hello! Yes, I know, it’s been too long...
