Assessment of Brain White Matter Fiber Bundle Atrophy in Patients with Friedreich Ataxia

Radiology, 255, 882-889. June 2010, doi: 10.1148/radiol.10091742
  
Elisabetta Pagani, MPhil, Andrea Ginestroni, MD,Riccardo Della Nave, MD, PhD,Federica Agosta, MD,Fabrizio Salvi, MD,Giuseppe De Michele, MD,Silvia Piacentini, MD,Massimo Filippi, MD and Mario Mascalchi, MD, PhD

Mitochondrial iron trafficking and the integration of iron metabolism between the mitochondrion and cytosol.

Proc Natl Acad Sci U S A. 2010 May 21.

Richardson DR, Lane DJ, Becker EM, Huang ML, Whitnall M, Rahmanto YS, Sheftel AD, Ponka P.

Iron Metabolism and Chelation Program, Discipline of Pathology, University of Sydney, NSW 2006, Australia.

Repligen Receives Orphan Drug Designation from the FDA for RG2833 for Friedreich's Ataxia

WALTHAM, Mass., May 24 /PRNewswire-FirstCall/ -- Repligen Corporation (Nasdaq: RGEN) announced today that the Office of Orphan Products Development of the Food and Drug Administration (FDA) has granted orphan drug designation to RG2833, a selective histone deacetylase 3 (HDAC-3) inhibitor for the treatment of Friedreich's ataxia.  Orphan drug designation qualifies Repligen to receive seven years of marketing exclusivity in the United States if the company is the first to obtain marketing approval for RG2833 for the treatment of Friedreich's ataxia.  This designation may also qualify Repligen to benefit from certain tax credits and a waiver of the company's obligation to pay the FDA application user fees for this product as required by the Prescription Drug User Fee Act. The U.S. Orphan Drug Act provides incentives for companies developing and marketing therapies for rare diseases, defined as those affecting fewer than 200,000 Americans.  There are approximately 15,000 people worldwide.......Read more

Iron Regulatory Proteins: From Molecular Mechanisms to Drug Development

Antioxidants & Redox Signaling. -Not available-, ahead of print. doi:10.1089/ars.2009.2983.

Stefania Recalcati,¹ Giorgio Minotti,² and Gaetano Cairo¹

¹Department of Human Morphology and Biomedical Sciences Città Studi, University of Milan, Milano, Italy.

²CIR and Drug Sciences, University Campus Bio-Medico and Fondazione Alberto Sordi-Research Institute on Aging, Roma, Italy.

Intracellular Redox Compartments: Mechanisms and Significances

Antioxidants & Redox Signaling. Ahead of print. doi:10.1089/ars.2009.3001.
Online Ahead of Print: May 17, 2010

Thomas Kietzmann

Department Chemistry/Biochemistry, University of Kaiserslautern, Kaiserslautern, Germany, and Department of Biochemistry, University of Oulu, Oulu, Finland.

Keywords:  reactive oxygen species (ROS),   act as signalling molecules,   hormones, growth factors, mechanical or chemical stress, mechanism of intracellular communication.

Human iron-sulfur cluster assembly, cellular iron homeostasis and disease.

Biochemistry. 2010 May 18. [Epub ahead of print]

Ye H, Rouault TA.

 Keyword: Iron sulfur (Fe-S) proteins, iron atoms, sulfur ligands, redox activity, enzymatic function, aconitase, succinate dehydrogenase, cellular iron homeostasis,  frataxin, GLRX5, ISCU, ABCB7, Friedreich's ataxia.

FULL TEXT

Santhera’s MICONOS Trial with Catena®/Sovrima® in Friedreich’s Ataxia Misses Primary Endpoint

Liestal, Switzerland, May 20, 2010 – Santhera Pharmaceuticals (SIX: SANN) announced today that its MICONOS Phase III study evaluating Catena®/Sovrima® for the treatment of Frie-dreich’s Ataxia missed its primary endpoint. Trends towards improvement in the key neuro-logical endpoint were however identified by a meta-analysis of all Santhera’s Phase II and III studies in the same indication. MICONOS also confirmed that Catena®/Sovrima® is safe and well tolerated at doses of up to 2250 mg/day.

Pregnancy with Friedreich ataxia: a retrospective review of medical risks and psychosocial implications.

Am J Obstet Gynecol. 2010 May 15

Friedman LS, Paulsen EK, Schadt KA, Brigatti KW, Driscoll DA, Farmer JM, Lynch DR.

Departments of Neurology and Pediatrics, University of Pennsylvania School of Medicine, and Children's Hospital of Philadelphia.

Keywords: Friedreich ataxia (FRDA), pregnancy, spontaneous abortion, preeclampsia, preterm birth.

Neuroprotective Multifunctional Iron Chelators: From Redox-Sensitive Process to Novel Therapeutic Opportunities

Antioxidants & Redox Signaling. Ahead of print. doi:10.1089/ars.2009.2929.
Online Ahead of Print: May 17, 2010

Orly Weinreb, Tamar Amit, Silvia Mandel, Lana Kupershmidt, Moussa B.H. Youdim. 
Eve Topf Centers of Excellence for Neurodegenerative Diseases Research and Department of Pharmacology, Rappaport Family Research Institute, Technion-Faculty of Medicine, Haifa, Israel.

Keywords:  cytotoxic signals,   neurodegenerative brain, oxidative stress, inflammation, accumulation of iron,  iron chelators,  hydroxyl radical formation,  Fenton reaction, hypoxia-inducible factor-1α (HIF-1α), prolyl-4-hydroxylases (PHDs).

Potential Friedreich's Ataxia Drug Based on Gottesfeld Lab Work Poised to Start Clinical Trials

 NEWS & WIEW, Online weekly of the Scripps Research Institute, Vol 10. Issue 17 / May 17, 2010

"I'm as pleased as can be," said Gottesfeld. "It's wonderful news that our lab's work could be launching human trials for Friedreich's ataxia."