Positive Preclinical Proof-of-Concept Results of BB-FA in Mice Model of Friedreich’s Ataxia

Positive Preclinical Proof-of-Concept Results of BB-FA in Mice Model of Friedreich’s Ataxia. BioBlast Announced Preclinical Data and Pipeline Updates. April 14, 2015

BioBlast (NasdaqGM: ORPN) announced positive in vitro and in vivo results from a therapy for Friedreich’s ataxia based on the mitochondria-targeting platform, providing support for further clinical development.

Emerging functional cross-talk between the Keap1-Nrf2 system and mitochondria

Emerging functional cross-talk between the Keap1-Nrf2 system and mitochondria. Ken Itoh,, Peng Ye, Tomoh Matsumiya, Kunikazu Tanji, and Taku Ozaki; J Clin Biochem Nutr. 2015 March; 56(2): 91–97. doi: 10.3164/jcbn.14-134

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DNA Topological and Epigenetic Regulation in Neurodevelopment and Disorders

DNA TOPOLOGICAL AND EPIGENETIC REGULATION IN NEURODEVELOPMENT AND DISORDERS . NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE, Project Number: 1K99NS091508-01, Project Leader: TEE, WEE WE, NEW YORK UNIVERSITY SCHOOL OF MEDICINE

PUBLIC HEALTH RELEVANCE: The goal of this project is to gain a mechanistic perspective on the roles of DNA topology and epigenetic regulation in neurodevelopment, and their implications in human neuropsychiatric disorders such as autism, and neurodegenerative diseases such as Freidreich's ataxia.

Differential Diagnosis of Sporadic Adult-Onset Ataxia: The Role of REM Sleep Behavior Disorder

Differential Diagnosis of Sporadic Adult-Onset Ataxia: The Role of REM Sleep Behavior Disorder. Hélio A.G. Teive, Walter O. Arruda, Adriana Moro, Mariana Moscovich, Renato P. Munhoz, Parkinsonism & Related Disorders, Available online 11 April 2015, ISSN 1353-8020, http://dx.doi.org/10.1016/j.parkreldis.2015.04.001.

Mitochondrial dynamism and heart disease: changing shape and shaping change

Mitochondrial dynamism and heart disease: changing shape and shaping change. Gerald W Dorn; EMBO Molecular Medicine
Early View, Article first published online: 10 APR 2015 DOI: 10.15252/emmm.201404575

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Horizon Pharma plc Receives FDA Fast Track Designation for ACTIMMUNE(R) in the Treatment of Friedreich's Ataxia

Horizon Pharma plc Receives FDA Fast Track Designation for ACTIMMUNE(R) in the Treatment of Friedreich's Ataxia . DUBLIN, IRELAND, Apr 10, 2015 (Marketwired via COMTEX)

The Company submitted the Investigational New Drug (IND) application for ACTIMMUNE in the treatment of FA in February 2015 and announced plans to begin a Phase 3 study in the second quarter in collaboration with the Friedreich's Ataxia Research Alliance (FARA) and a sub-set of FARA's Collaborative Clinical Research Network (CCRN) study centers in FA.

An open-label trial in Friedreich ataxia suggests clinical benefit with high-dose resveratrol, without effect on frataxin levels

An open-label trial in Friedreich ataxia suggests clinical benefit with high-dose resveratrol, without effect on frataxin levels. Eppie M. Yiu, Geneieve Tai, Roger E. Peverill, Katherine J. Lee, Kevin D. Croft, Trevor A. Mori, Barbara Scheiber-Mojdehkar, Brigitte Sturm, Monika Praschberger, Adam P. Vogel, Gary Rance, Sarah E. M. Stephenson, Joseph P. Sarsero, Creina Stockley, Chung-Yung J. Lee, Andrew Churchyard, Marguerite V. Evans-Galea, Monique M. Ryan, Paul J. Lockhart, Louise A. Corben, Martin B. Delatycki; Journal of Neurology, April 2015 DOI 10.1007/s00415-015-7719-2

Effect of mental fatigue on speed-accuracy trade-off

Effect of mental fatigue on speed-accuracy trade-off. Vianney Rozand, Florent Lebon, Charalambos Papaxanthis, Romuald Lepers, Neuroscience, Available online 4 April 2015, ISSN 0306-4522, http://dx.doi.org/10.1016/j.neuroscience.2015.03.066.

Previous studies suggested that the speed-accuracy trade-off is altered in specific cases, e.g., the patients with Friedreich ataxia that alter motor coordination but not cognitive functions (Corben et al., 2011) or the elderly (Sleimen-Malkoun et al., 2013). In these populations, movement duration was slowed compared to healthy young subjects, especially for higher difficulties.

Autophagy in neurodegenerative diseases: from pathogenic dysfunction to therapeutic modulation

Autophagy in neurodegenerative diseases: from pathogenic dysfunction to therapeutic modulation. Marta Martinez-Vicente, Seminars in Cell & Developmental Biology, Available online 2 April 2015, ISSN 1084-9521, http://dx.doi.org/10.1016/j.semcdb.2015.03.005.

Autophagic dysfunction has been described in many neurodegenerative diseases, ranging from Alzheimer’s disease (AD), to Parkinson’s disease (PD), Huntington’s disease (HD), amyotrophic lateral sclerosis (ALS), Friedreich's ataxia and others. Failure of the autophagic process may thus be one of the factors contributing to neuronal cell death

Risk of Heart Failure and Death Among Patients With Friedreich Ataxia Admitted for Non-Cardiac Etiologies

Risk of Heart Failure and Death Among Patients With Friedreich Ataxia Admitted for Non-Cardiac Etiologies. K.Y. Lin, D. Lynch, M.J. O’Connor, J. Rossano, The Journal of Heart and Lung Transplantation, Volume 34, Issue 4, Supplement, April 2015, Page S186, ISSN 1053-2498, http://dx.doi.org/10.1016/j.healun.2015.01.508.