Gene editing of human hematopoietic stem and progenitor cells for Friedreich’s ataxia using CRISPR/Cas9 technology

  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...

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New Collaborative Clinical Research Site on Friedreich’s Ataxia in Montreal

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)...

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Reata Announces Positive Results of Omaveloxolone for Friedreich’s Ataxia

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...

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MINORYX THERAPEUTICS COMPLETES ENROLLMENT IN FRAMES PHASE 2 TRIAL WITH LERIGLITAZONE IN FRIEDREICH’S ATAXIA

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...

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New developments in pharmacotherapy for Friedreich ataxia article

Current and emerging therapies for Friedrich’s Ataxia (FA) focus on reversing the deleterious effects of such deficiency including mitochondrial augmentation and increasing frataxin levels, providing the possibility of treatment options for this physiologically complex, multisystem disorder. In this review article, the authors discuss the current and prior in vivo and...

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