Horizon Pharma plc Announces Presentation of ACTIMMUNE(R) Phase 2 Data in Friedreich's Ataxia

Horizon Pharma plc Announces Presentation of ACTIMMUNE(R) Phase 2 Data in Friedreich's Ataxia. DUBLIN, IRELAND -- (Marketwired) -- 10/09/14

Positive Clinical Efficacy Signal Seen in Study of Twelve Patients; Company Planning Phase 3 Program

An abstract of the data has been published in a supplement to the Annals of Neurology and is being presented as a poster during the 139th Annual Meeting of the American Neurological Association in Baltimore, MD on Monday, October 13.

The roles of glutaredoxins ligating Fe–S clusters: Sensing, transfer or repair functions?

The roles of glutaredoxins ligating Fe–S clusters: Sensing, transfer or repair functions?; Jérémy Couturier, Jonathan Przybyla-Toscano, Thomas Roret, Claude Didierjean, Nicolas Rouhier, Biochimica et Biophysica Acta (BBA) - Molecular Cell Research, Available online 28 September 2014, ISSN 0167-4889, http://dx.doi.org/10.1016/j.bbamcr.2014.09.018.

Trinucleotide expansion in disease: why is there a length threshold?

Trinucleotide expansion in disease: why is there a length threshold?. Do-Yup Lee, Cynthia T. McMurray; Current Opinion in Genetics & Development 2014, 26:131–140 DOI: http://dx.doi.org/10.1016/j.gde.2014.07.003

Horizon Pharma plc Receives Orphan-Drug Designation for ACTIMMUNE(R) (Interferon gamma-1b) in Friedreich's Ataxia

Horizon Pharma plc Receives Orphan-Drug Designation for ACTIMMUNE(R) (Interferon gamma-1b) in Friedreich's Ataxia. Oct 03, 2014 (Marketwired via COMTEX).

RTA 408 Capsules in Patients With Friedreich's Ataxia (MOXIe)

RTA 408 Capsules in Patients With Friedreich's Ataxia (MOXIe). A Phase 2 Study of the Safety, Efficacy, and Pharmacodynamics of RTA 408 in the Treatment of Friedreich's Ataxia (MOXIe). ClinicalTrials.gov Identifier: NCT02255435

Reata Announces the Initiation of Phase 2 Studies Examining RTA 408 for the Treatment of Friedreich's Ataxia and Mitochondrial Myopathies

Reata Announces the Initiation of Phase 2 Studies Examining RTA 408 for the Treatment of Friedreich's Ataxia and Mitochondrial Myopathies. -(Marketwired - September 30, 2014), SOURCE: Reata Pharmaceuticals, Inc.

The two initial Phase 2 trials will both be multi-center, double-blind, randomized, dose-ranging, placebo-controlled studies.

Comparing speech characteristics in spinocerebellar ataxias type 3 and type 6 with Friedreich ataxia

Comparing speech characteristics in spinocerebellar ataxias type 3 and type 6 with Friedreich ataxia. Bettina Brendel, Matthis Synofzik, Hermann Ackermann, Tobias Lindig, Theresa Schölderle, Ludger Schöls, Wolfram Ziegler; Journal of Neurology, September 2014. DOI 10.1007/s00415-014-7511-8

Idebenone and neuroprotection: antioxidant, pro-oxidant, or electron carrier?

Idebenone and neuroprotection: antioxidant, pro-oxidant, or electron carrier?. Sausan Jaber, Brian M. Polster; Journal of Bioenergetics and Biomembranes
September 2014 DOI 10.1007/s10863-014-9571-y

A Novel Mouse Model for Friedreich's Ataxia

A Novel Mouse Model for Friedreich's Ataxia. Inventors: Geschwind, Daniel H.; UCLA Office of Intellectual Property & Industry Sponsored Research

This new model exhibits various symptoms observed in FRDA patients, including cardiac atrophy, elevated iron-responsive proteins, neurodegeneration, motor neuropathy–and for the first time in an FRDA model–scoliosis and ataxia.

Next-Generation R&D Partnerships: The NCATS Success Story

Next-Generation R&D Partnerships: The NCATS Success Story , Information Technology and Innovation Foundation.

Free event on Wednesday, October 29, 2014,10:00 AM - 11:30 AM, Rayburn House Office Building 45 Independence Ave SW
(2168) Washington, DC20515