FA & ACTIMMUNE Phase 2 Clinical Trial Results

FA & ACTIMMUNE Phase 2 Clinical Trial Results. Friedreich's Ataxia Analyst Day Presentation, Horizon Pharma plc, October 13, 2014

Clinical Potentials of Cardiomyocytes Derived from Patient-Specific Induced Pluripotent Stem Cells

Clinical Potentials of Cardiomyocytes Derived from Patient-Specific Induced Pluripotent Stem Cells. Kwong-Man Ng, Cheuk-Yiu Law and Hung-Fat Tse; J. Clin. Med. 2014, 3(4), 1105-1123; doi:10.3390/jcm3041105

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The cardiomyocytes derived from patients-specific iPSCs are of great potential in many clinical applications. This authentic human cardiomyocyte-based system is expected to compensate for the limitations of the current experimental animal models. This review provides detailed descriptions in the strategies and workflow of using the patient-specific iPSCs-derived cardiomyocytes in regenerative medicine, disease modeling and pharmacological applications. The examples illustrated in this review clearly evidenced the practical values of this novel technology. However, various limitations, such as the immaturities of iPSCs-derived cardiomyocytes, still need to be addressed, and future studies resolving these issues would be beneficial to the use of patient-specific iPSCs in clinical applications.

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

Cardiac Manifestations of Friedreich Ataxia

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

Cardiac Manifestations of Friedreich Ataxia from Hari Krishnan

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

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