Monday, October 13, 2014

Horizon Pharma plc Reviews Future ACTIMMUNE® Opportunities at Analyst Day

Horizon Pharma plc Reviews Future ACTIMMUNE® Opportunities at Analyst Day. KING OF PRUSSIA, PA--(Marketwired - Oct 13, 2014)

SOURCE: Horizon Pharma plc

Sunday, October 12, 2014

Cardiac Manifestations of Friedreich Ataxia

Cardiac Manifestations of Friedreich Ataxia. Hari Krishnan NAIR, Cardiology Boston Children's Hospital, Published on October 12, 2014

Saturday, October 11, 2014

The Diabetes Drug Target MitoNEET Governs a Novel Trafficking Pathway to Rebuild an Fe-S Cluster into Cytosolic Aconitase/Iron Regulatory Protein 1

The Diabetes Drug Target MitoNEET Governs a Novel Trafficking Pathway to Rebuild an Fe-S Cluster into Cytosolic Aconitase/Iron Regulatory Protein 1 . Ioana Ferecatu, Sergio Gonçalves, Marie-Pierre Golinelli-Cohen, Martin Clémancey, Alain Martelli, Sylvie Riquier, Eric Guittet, Jean-Marc Latour, Hélène Puccio, Jean-Claude Drapier, Ewen Lescop and Cécile Bouton; J. Biol. Chem. jbc.M114.548438. 2014, doi:10.1074/jbc.M114.548438

Friday, October 10, 2014

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.

Sunday, October 5, 2014

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).


Friday, October 3, 2014

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

Wednesday, October 1, 2014

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.

Monday, September 29, 2014

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