Friedreich's Ataxia

News Friedreich's Ataxia

• Santhera Presents Positive Data from IONIA Open-Label Extension Study in Friedreich's Ataxia

  Santhera Pharmaceuticals announced that it will present positive data from an open-label extension study (IONIA-E) evaluating Catena® for the treatment of Friedreich's Ataxia. The findings indicate that Catena® can offer therapeutic benefit to pediatric patients by improving overall neurological fun more

• Santhera's MICONOS Trial with Catena/Sovrima in Friedreich's Ataxia Misses Primary Endpoint

  Santhera Pharmaceuticals announced that its MICONOS Phase III study evaluating Catena®/Sovrima® for the treatment of Friedreich's Ataxia missed its primary endpoint. Trends towards improvement in the key neurological endpoint were however identified by a meta-analysis of all Santhera's Phase II and more

• Lundbeck starts clinical phase IIa with Lu AA24493 (cEPO) in Friedreich's ataxia

  H. Lundbeck A/S strengthens its pipeline of pharmaceuticals in clinical development by initiating phase IIa clinical studies with the innovative project Lu AA24493 in order to evaluate safety and tolerability and to explore theoretical efficacy parameters of the drug in humans. Lundbeck expects to e more

Publications Friedreich's Ataxia

• Triple therapy with deferiprone, idebenone and riboflavin in Friedreich's ataxia – open‐label trial

  Objectives The objective of the study was to test the efficacy, safety and tolerability of triple therapy with deferiprone, idebenone and riboflavin in Friedreich's ataxia (FRDA) patients in a clinical pilot study. Patients and methods Patients included in this study were 10 males and thr more

• Identification of nonferritin mitochondrial iron deposits in a mouse model of Friedreich ataxia [Medical Sciences]

  There is no effective treatment for the cardiomyopathy of the most common autosomal recessive ataxia, Friedreich ataxia (FA). This disease is due to decreased expression of the mitochondrial protein, frataxin, which leads to alterations in mitochondrial iron (Fe) metabolism. The identification of po more

• Apn1 AP-endonuclease is essential for the repair of oxidatively damaged DNA bases in yeast frataxin-deficient cells

  Frataxin deficiency results in mitochondrial dysfunction and oxidative stress and it is the cause of the hereditary neurodegenerative disease Friedreich ataxia (FA). Here, we present evidence that one of the pleiotropic effects of oxidative stress in frataxin-deficient yeast cells (yfh1 mutant) is more

Market studies Friedreich's Ataxia

• Friedreich Ataxia Global Clinical Trials Review, H1, 2012

  Introduction GlobalData's clinical trial report, “Friedreich Ataxia Global Clinical Trials Review, H2, 2011" provides data on the Friedreich Ataxia clinical trial scenario. This report provides elemental information and data relating to the clinical trials on Friedreich Ataxia. It includes an overv more

• Friedreich Ataxia – Pipeline Review, H1 2012

  Introduction Global Markets Direct’s, 'Friedreich Ataxia - Pipeline Review, H1 2012', provides an overview of the Friedreich Ataxia therapeutic pipeline. This report provides information on the therapeutic development for Friedreich Ataxia , complete with latest updates, and special features on la more

• Repligen Corporation (RGEN) - Financial and Strategic SWOT Analysis Review

  Repligen Corporation (Repligen) is a publicly held research focused bio pharmaceutical company engaged in development of innovative therapeutics for neurological disorders. Repligen offers recombinant protein A, immobilized protein A and protein A ELISA kits under its marketed product portfolio. In more

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