Open-label pilot study of interferon gamma-1b in Friedreich ataxia
Objectives/Aims This is an open‐label trial of the safety of interferon gamma‐1b (IFN‐γ) and its effect on frataxin levels and neurologic measures in 12 children with Friedreich ataxia. Materials and Methods Interferon gamma‐1b was administered via subcutaneous injection three times weekly. The dose...
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| Published in | Acta neurologica Scandinavica Vol. 132; no. 1; pp. 7 - 15 |
|---|---|
| Main Authors | , , , , , , , , , , , |
| Format | Journal Article |
| Language | English |
| Published |
Denmark
Blackwell Publishing Ltd
01.07.2015
John Wiley & Sons, Inc |
| Subjects | |
| Online Access | Get full text |
| ISSN | 0001-6314 1600-0404 1600-0404 |
| DOI | 10.1111/ane.12337 |
Cover
| Abstract | Objectives/Aims
This is an open‐label trial of the safety of interferon gamma‐1b (IFN‐γ) and its effect on frataxin levels and neurologic measures in 12 children with Friedreich ataxia.
Materials and Methods
Interferon gamma‐1b was administered via subcutaneous injection three times weekly. The dose increased from 10 to 50 mcg/m2 during the first four weeks and then remained at 50 mcg/m2 for final eight weeks. Safety assessments included laboratory testing, electrocardiogram, and monitoring of adverse events. The primary efficacy outcome measure was frataxin level in whole blood. Secondary measures included frataxin levels in multiple tissues, frataxin mRNA levels, Friedreich Ataxia Rating Scale (FARS) scores and other neurologic evaluations. Statistical analyses were performed via SAS and STATA.
Results
Interferon gamma‐1b was well tolerated with no serious adverse events, and only two subjects reporting severe adverse events and subsequent dose reductions. Small but significant changes in frataxin levels were observed in red blood cells, PBMC, and platelets after 12 weeks of treatment. However, the magnitude of change was small and varied between tissues. Mean improvement in FARS score was equivalent to roughly 18 months of disease progression after 12 weeks of treatment (P = 0.008). No other statistically significant changes were observed. No statistically significant relationships were observed between frataxin protein levels, FARS scores, and in vivo IFN‐γ levels.
Conclusions
Interferon gamma‐1b improved FARS scores without a clear relationship to changes in frataxin levels. Larger, longer placebo‐controlled trials including biochemical assessments in affected tissues are necessary to evaluate fully the efficacy and utility of IFN‐γ in FRDA. |
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| AbstractList | Objectives/Aims This is an open-label trial of the safety of interferon gamma-1b (IFN- gamma ) and its effect on frataxin levels and neurologic measures in 12 children with Friedreich ataxia. Materials and Methods Interferon gamma-1b was administered via subcutaneous injection three times weekly. The dose increased from 10 to 50 mcg/m super(2) during the first four weeks and then remained at 50 mcg/m super(2) for final eight weeks. Safety assessments included laboratory testing, electrocardiogram, and monitoring of adverse events. The primary efficacy outcome measure was frataxin level in whole blood. Secondary measures included frataxin levels in multiple tissues, frataxin mRNA levels, Friedreich Ataxia Rating Scale (FARS) scores and other neurologic evaluations. Statistical analyses were performed via SAS and STATA. Results Interferon gamma-1b was well tolerated with no serious adverse events, and only two subjects reporting severe adverse events and subsequent dose reductions. Small but significant changes in frataxin levels were observed in red blood cells, PBMC, and platelets after 12 weeks of treatment. However, the magnitude of change was small and varied between tissues. Mean improvement in FARS score was equivalent to roughly 18 months of disease progression after 12 weeks of treatment (P = 0.008). No other statistically significant changes were observed. No statistically significant relationships were observed between frataxin protein levels, FARS scores, and in vivo IFN- gamma levels. Conclusions Interferon gamma-1b improved FARS scores without a clear relationship to changes in frataxin levels. Larger, longer placebo-controlled trials including biochemical assessments in affected tissues are necessary to evaluate fully the efficacy and utility of IFN- gamma in FRDA. This is an open-label trial of the safety of interferon gamma-1b (IFN-γ) and its effect on frataxin levels and neurologic measures in 12 children with Friedreich ataxia. Interferon gamma-1b was administered via subcutaneous injection three times weekly. The dose increased from 10 to 50 mcg/m(2) during the first four weeks and then remained at 50 mcg/m(2) for final eight weeks. Safety assessments included laboratory testing, electrocardiogram, and monitoring of adverse events. The primary efficacy outcome measure was frataxin level in whole blood. Secondary measures included frataxin levels in multiple tissues, frataxin mRNA levels, Friedreich Ataxia Rating Scale (FARS) scores and other neurologic evaluations. Statistical analyses were performed via SAS and STATA. Interferon gamma-1b was well tolerated with no serious adverse events, and only two subjects reporting severe adverse events and subsequent dose reductions. Small but significant changes in frataxin levels were observed in red blood cells, PBMC, and platelets after 12 weeks of treatment. However, the magnitude of change was small and varied between tissues. Mean improvement in FARS score was equivalent to roughly 18 months of disease progression after 12 weeks of treatment (P = 0.008). No other statistically significant changes were observed. No statistically significant relationships were observed between frataxin protein levels, FARS scores, and in vivo IFN-γ levels. Interferon gamma-1b improved FARS scores without a clear relationship to changes in frataxin levels. Larger, longer placebo-controlled trials including biochemical assessments in affected tissues are necessary to evaluate fully the efficacy and utility of IFN-γ in FRDA. Objectives/Aims This is an open-label trial of the safety of interferon gamma-1b (IFN-[gamma]) and its effect on frataxin levels and neurologic measures in 12 children with Friedreich ataxia. Materials and Methods Interferon gamma-1b was administered via subcutaneous injection three times weekly. The dose increased from 10 to 50 mcg/m2 during the first four weeks and then remained at 50 mcg/m2 for final eight weeks. Safety assessments included laboratory testing, electrocardiogram, and monitoring of adverse events. The primary efficacy outcome measure was frataxin level in whole blood. Secondary measures included frataxin levels in multiple tissues, frataxin mRNA levels, Friedreich Ataxia Rating Scale (FARS) scores and other neurologic evaluations. Statistical analyses were performed via SAS and STATA. Results Interferon gamma-1b was well tolerated with no serious adverse events, and only two subjects reporting severe adverse events and subsequent dose reductions. Small but significant changes in frataxin levels were observed in red blood cells, PBMC, and platelets after 12 weeks of treatment. However, the magnitude of change was small and varied between tissues. Mean improvement in FARS score was equivalent to roughly 18 months of disease progression after 12 weeks of treatment (P = 0.008). No other statistically significant changes were observed. No statistically significant relationships were observed between frataxin protein levels, FARS scores, and in vivoIFN-[gamma] levels. Conclusions Interferon gamma-1b improved FARS scores without a clear relationship to changes in frataxin levels. Larger, longer placebo-controlled trials including biochemical assessments in affected tissues are necessary to evaluate fully the efficacy and utility of IFN-[gamma] in FRDA. Objectives/Aims This is an open‐label trial of the safety of interferon gamma‐1b (IFN‐γ) and its effect on frataxin levels and neurologic measures in 12 children with Friedreich ataxia. Materials and Methods Interferon gamma‐1b was administered via subcutaneous injection three times weekly. The dose increased from 10 to 50 mcg/m2 during the first four weeks and then remained at 50 mcg/m2 for final eight weeks. Safety assessments included laboratory testing, electrocardiogram, and monitoring of adverse events. The primary efficacy outcome measure was frataxin level in whole blood. Secondary measures included frataxin levels in multiple tissues, frataxin mRNA levels, Friedreich Ataxia Rating Scale (FARS) scores and other neurologic evaluations. Statistical analyses were performed via SAS and STATA. Results Interferon gamma‐1b was well tolerated with no serious adverse events, and only two subjects reporting severe adverse events and subsequent dose reductions. Small but significant changes in frataxin levels were observed in red blood cells, PBMC, and platelets after 12 weeks of treatment. However, the magnitude of change was small and varied between tissues. Mean improvement in FARS score was equivalent to roughly 18 months of disease progression after 12 weeks of treatment (P = 0.008). No other statistically significant changes were observed. No statistically significant relationships were observed between frataxin protein levels, FARS scores, and in vivo IFN‐γ levels. Conclusions Interferon gamma‐1b improved FARS scores without a clear relationship to changes in frataxin levels. Larger, longer placebo‐controlled trials including biochemical assessments in affected tissues are necessary to evaluate fully the efficacy and utility of IFN‐γ in FRDA. This is an open-label trial of the safety of interferon gamma-1b (IFN-γ) and its effect on frataxin levels and neurologic measures in 12 children with Friedreich ataxia.OBJECTIVES/AIMSThis is an open-label trial of the safety of interferon gamma-1b (IFN-γ) and its effect on frataxin levels and neurologic measures in 12 children with Friedreich ataxia.Interferon gamma-1b was administered via subcutaneous injection three times weekly. The dose increased from 10 to 50 mcg/m(2) during the first four weeks and then remained at 50 mcg/m(2) for final eight weeks. Safety assessments included laboratory testing, electrocardiogram, and monitoring of adverse events. The primary efficacy outcome measure was frataxin level in whole blood. Secondary measures included frataxin levels in multiple tissues, frataxin mRNA levels, Friedreich Ataxia Rating Scale (FARS) scores and other neurologic evaluations. Statistical analyses were performed via SAS and STATA.MATERIALS AND METHODSInterferon gamma-1b was administered via subcutaneous injection three times weekly. The dose increased from 10 to 50 mcg/m(2) during the first four weeks and then remained at 50 mcg/m(2) for final eight weeks. Safety assessments included laboratory testing, electrocardiogram, and monitoring of adverse events. The primary efficacy outcome measure was frataxin level in whole blood. Secondary measures included frataxin levels in multiple tissues, frataxin mRNA levels, Friedreich Ataxia Rating Scale (FARS) scores and other neurologic evaluations. Statistical analyses were performed via SAS and STATA.Interferon gamma-1b was well tolerated with no serious adverse events, and only two subjects reporting severe adverse events and subsequent dose reductions. Small but significant changes in frataxin levels were observed in red blood cells, PBMC, and platelets after 12 weeks of treatment. However, the magnitude of change was small and varied between tissues. Mean improvement in FARS score was equivalent to roughly 18 months of disease progression after 12 weeks of treatment (P = 0.008). No other statistically significant changes were observed. No statistically significant relationships were observed between frataxin protein levels, FARS scores, and in vivo IFN-γ levels.RESULTSInterferon gamma-1b was well tolerated with no serious adverse events, and only two subjects reporting severe adverse events and subsequent dose reductions. Small but significant changes in frataxin levels were observed in red blood cells, PBMC, and platelets after 12 weeks of treatment. However, the magnitude of change was small and varied between tissues. Mean improvement in FARS score was equivalent to roughly 18 months of disease progression after 12 weeks of treatment (P = 0.008). No other statistically significant changes were observed. No statistically significant relationships were observed between frataxin protein levels, FARS scores, and in vivo IFN-γ levels.Interferon gamma-1b improved FARS scores without a clear relationship to changes in frataxin levels. Larger, longer placebo-controlled trials including biochemical assessments in affected tissues are necessary to evaluate fully the efficacy and utility of IFN-γ in FRDA.CONCLUSIONSInterferon gamma-1b improved FARS scores without a clear relationship to changes in frataxin levels. Larger, longer placebo-controlled trials including biochemical assessments in affected tissues are necessary to evaluate fully the efficacy and utility of IFN-γ in FRDA. |
| Author | Strawser, C. Brocht, A. Wilson, R. B. Dong, Y. Seyer, L. Cotticelli, M. G. Greeley, N. Schadt, K. Foerster, D. Gelbard, S. Lynch, D. R. Farmer, J. |
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| BackLink | https://www.ncbi.nlm.nih.gov/pubmed/25335475$$D View this record in MEDLINE/PubMed |
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| References | Babcock M, de Silva D, Oaks R et al. Regulation of mitochondrial iron accumulation by Yfh1p, a putative homolog of frataxin. Science 1997;276:1709-12. Schadt K, Friedman LS, Regner SR, Mark GE, Lynch DR, Lin KY. Cross-sectional analysis of electrocardiograms in a large heterogeneous cohort of Friedreich ataxia subjects. J Child Neurol 2012;27:1187-92. Lynch DR, Perlman SL, Meier T. A phase 3, double-blind, placebo-controlled trial of idebenone in Friedreich ataxia. Arch Neurol 2010;67:941-7. Rai M, Soragni E, Jenssen K et al. HDAC inhibitors correct frataxin deficiency in a Friedreich ataxia mouse model. PLoS ONE 2008;3:e1958. Deutsch EC, Santani AB, Perlman SL et al. A rapid, noninvasive immunoassay for frataxin: utility in assessment of Friedreich ataxia. Mol Genet Metab 2010;101:238-45. Harding AE. Friedreich's ataxia: a clinical and genetic study of 90 families with an analysis of early diagnostic criteria and intrafamilial clustering of clinical features. Brain 1981;104:589-620. Sandi C, Pinto RM, Al-Mahdawi S et al. Prolonged treatment with pimelico-aminobenzamide HDAC inhibitors ameliorates the disease phenotype of a Friedreich ataxia mouse model. Neurobiol Dis 2011;42:496-505. Lynch DR, Willi SM, Wilson RB et al. A0001 in Friedreich ataxia: biochemical characterization and effects in a clinical trial. Mov Disord 2012;27:1026-33. Subramony SH, May W, Lynch D et al. Cooperative ataxia group measuring Friedreich ataxia: interrater reliability of a neurologic rating scale. Neurology 2005;64:1261-2. Rance G, Ryan MM, Carew P et al. Binaural speech processing in individuals with auditory neuropathy. Neuroscience 2012;226:227-35. Simon D, Seznec H, Gansmuller A et al. Friedreich ataxia mouse models with progressive cerebellar and sensory ataxia reveal autophagic neurodegeneration in dorsal root ganglia. J Neurosci 2004;24:1987-95. Lynch DR, Farmer JM, Balcer LJ, Wilson RB. Friedreich ataxia: effects of genetic understanding on clinical evaluation and therapy. Arch Neurol 2002;59:743-7. Boesch S, Nachbauer W, Mariotti C et al. Safety and tolerability of carbamylated erythropoietin in Friedreich's ataxia. Mov Disord 2014;29:935-9. Lynch DR, Farmer JM, Tsou AY et al. Measuring Friedreich ataxia: complementary features of examination performance measures. Neurology 2006;66:1711-6. Rötig A, de Lonlay P, Chretien D et al. Aconitase and mitochondrial iron-sulphur protein deficiency in Friedreich ataxia. Nat Genet 1997;17:215-7. Arpa J, Sanz-Gallego I, Rodriguez-de-Rivera FJ et al. Triple therapy with deferiprone, Idebenone, and riboflavin in Friedreich's ataxia - open-label trial. Acta Neurol Scand 2014;129:32-40. Friedman LS, Farmer JM, Perlman S et al. Measuring the rate of progression in Friedreich ataxia: implications for clinical trial design. Mov Disord 2010;25:426-32. Devane JG, Martin ML, Matson MA. A short 2 week dose titration regimen reduces the severity of flu-like symptoms with initial interferon gamma-1b treatment. Curr Med Res Opin 2014;30:1179-87. Koeppen AH. Friedreich's ataxia: pathology, pathogenesis, and molecular genetics. J Neurol Sci 2011;303:1-12. Tomassini B, Arcuri G, Fortuni S et al. Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model. Hum Mol Genet 2012;21:2855-61. Mariotti C, Fancellu R, Caldarazzo S et al. Erythropoietin in Friedreich ataxia: no effect on frataxin in a randomized controlled trial. Mov Disord 2012;27:446-9. 2011; 303 2010; 67 2014; 129 2002; 59 2010; 25 1981; 104 2006; 66 1997; 276 2010; 101 2004; 24 2011; 42 1997; 17 2005; 64 2012; 27 2014; 29 2008; 3 2014; 30 2012; 226 2012; 21 Boesch (10.1111/ane.12337-BIB0011|ane12337-cit-0011) 2014; 29 Tomassini (10.1111/ane.12337-BIB0015|ane12337-cit-0015) 2012; 21 Harding (10.1111/ane.12337-BIB0001|ane12337-cit-0001) 1981; 104 Friedman (10.1111/ane.12337-BIB0018|ane12337-cit-0018) 2010; 25 Lynch (10.1111/ane.12337-BIB0009|ane12337-cit-0009) 2012; 27 Mariotti (10.1111/ane.12337-BIB0012|ane12337-cit-0012) 2012; 27 Lynch (10.1111/ane.12337-BIB0002|ane12337-cit-0002) 2002; 59 Arpa (10.1111/ane.12337-BIB0010|ane12337-cit-0010) 2014; 129 Lynch (10.1111/ane.12337-BIB0008|ane12337-cit-0008) 2010; 67 Devane (10.1111/ane.12337-BIB0021|ane12337-cit-0021) 2014; 30 Rai (10.1111/ane.12337-BIB0013|ane12337-cit-0013) 2008; 3 Sandi (10.1111/ane.12337-BIB0014|ane12337-cit-0014) 2011; 42 Subramony (10.1111/ane.12337-BIB0017|ane12337-cit-0017) 2005; 64 Deutsch (10.1111/ane.12337-BIB0007|ane12337-cit-0007) 2010; 101 Schadt (10.1111/ane.12337-BIB0020|ane12337-cit-0020) 2012; 27 Koeppen (10.1111/ane.12337-BIB0006|ane12337-cit-0006) 2011; 303 Lynch (10.1111/ane.12337-BIB0016|ane12337-cit-0016) 2006; 66 Babcock (10.1111/ane.12337-BIB0004|ane12337-cit-0004) 1997; 276 Rötig (10.1111/ane.12337-BIB0005|ane12337-cit-0005) 1997; 17 Rance (10.1111/ane.12337-BIB0019|ane12337-cit-0019) 2012; 226 Simon (10.1111/ane.12337-BIB0003|ane12337-cit-0003) 2004; 24 |
| References_xml | – reference: Schadt K, Friedman LS, Regner SR, Mark GE, Lynch DR, Lin KY. Cross-sectional analysis of electrocardiograms in a large heterogeneous cohort of Friedreich ataxia subjects. J Child Neurol 2012;27:1187-92. – reference: Lynch DR, Perlman SL, Meier T. A phase 3, double-blind, placebo-controlled trial of idebenone in Friedreich ataxia. Arch Neurol 2010;67:941-7. – reference: Rai M, Soragni E, Jenssen K et al. HDAC inhibitors correct frataxin deficiency in a Friedreich ataxia mouse model. PLoS ONE 2008;3:e1958. – reference: Lynch DR, Farmer JM, Tsou AY et al. Measuring Friedreich ataxia: complementary features of examination performance measures. Neurology 2006;66:1711-6. – reference: Subramony SH, May W, Lynch D et al. Cooperative ataxia group measuring Friedreich ataxia: interrater reliability of a neurologic rating scale. Neurology 2005;64:1261-2. – reference: Simon D, Seznec H, Gansmuller A et al. Friedreich ataxia mouse models with progressive cerebellar and sensory ataxia reveal autophagic neurodegeneration in dorsal root ganglia. J Neurosci 2004;24:1987-95. – reference: Harding AE. Friedreich's ataxia: a clinical and genetic study of 90 families with an analysis of early diagnostic criteria and intrafamilial clustering of clinical features. Brain 1981;104:589-620. – reference: Rötig A, de Lonlay P, Chretien D et al. Aconitase and mitochondrial iron-sulphur protein deficiency in Friedreich ataxia. Nat Genet 1997;17:215-7. – reference: Arpa J, Sanz-Gallego I, Rodriguez-de-Rivera FJ et al. Triple therapy with deferiprone, Idebenone, and riboflavin in Friedreich's ataxia - open-label trial. Acta Neurol Scand 2014;129:32-40. – reference: Babcock M, de Silva D, Oaks R et al. Regulation of mitochondrial iron accumulation by Yfh1p, a putative homolog of frataxin. Science 1997;276:1709-12. – reference: Mariotti C, Fancellu R, Caldarazzo S et al. Erythropoietin in Friedreich ataxia: no effect on frataxin in a randomized controlled trial. Mov Disord 2012;27:446-9. – reference: Rance G, Ryan MM, Carew P et al. Binaural speech processing in individuals with auditory neuropathy. Neuroscience 2012;226:227-35. – reference: Deutsch EC, Santani AB, Perlman SL et al. A rapid, noninvasive immunoassay for frataxin: utility in assessment of Friedreich ataxia. Mol Genet Metab 2010;101:238-45. – reference: Lynch DR, Willi SM, Wilson RB et al. A0001 in Friedreich ataxia: biochemical characterization and effects in a clinical trial. Mov Disord 2012;27:1026-33. – reference: Boesch S, Nachbauer W, Mariotti C et al. Safety and tolerability of carbamylated erythropoietin in Friedreich's ataxia. Mov Disord 2014;29:935-9. – reference: Sandi C, Pinto RM, Al-Mahdawi S et al. Prolonged treatment with pimelico-aminobenzamide HDAC inhibitors ameliorates the disease phenotype of a Friedreich ataxia mouse model. Neurobiol Dis 2011;42:496-505. – reference: Friedman LS, Farmer JM, Perlman S et al. Measuring the rate of progression in Friedreich ataxia: implications for clinical trial design. Mov Disord 2010;25:426-32. – reference: Tomassini B, Arcuri G, Fortuni S et al. Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model. Hum Mol Genet 2012;21:2855-61. – reference: Devane JG, Martin ML, Matson MA. A short 2 week dose titration regimen reduces the severity of flu-like symptoms with initial interferon gamma-1b treatment. Curr Med Res Opin 2014;30:1179-87. – reference: Lynch DR, Farmer JM, Balcer LJ, Wilson RB. Friedreich ataxia: effects of genetic understanding on clinical evaluation and therapy. Arch Neurol 2002;59:743-7. – reference: Koeppen AH. Friedreich's ataxia: pathology, pathogenesis, and molecular genetics. J Neurol Sci 2011;303:1-12. – volume: 129 start-page: 32 year: 2014 end-page: 40 article-title: Triple therapy with deferiprone, Idebenone, and riboflavin in Friedreich's ataxia – open‐label trial publication-title: Acta Neurol Scand – volume: 226 start-page: 227 year: 2012 end-page: 35 article-title: Binaural speech processing in individuals with auditory neuropathy publication-title: Neuroscience – volume: 27 start-page: 1026 year: 2012 end-page: 33 article-title: A0001 in Friedreich ataxia: biochemical characterization and effects in a clinical trial publication-title: Mov Disord – volume: 17 start-page: 215 year: 1997 end-page: 7 article-title: Aconitase and mitochondrial iron‐sulphur protein deficiency in Friedreich ataxia publication-title: Nat Genet – volume: 64 start-page: 1261 year: 2005 end-page: 2 article-title: Cooperative ataxia group measuring Friedreich ataxia: interrater reliability of a neurologic rating scale publication-title: Neurology – volume: 101 start-page: 238 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– volume: 27 start-page: 446 year: 2012 end-page: 9 article-title: Erythropoietin in Friedreich ataxia: no effect on frataxin in a randomized controlled trial publication-title: Mov Disord – volume: 30 start-page: 1179 year: 2014 end-page: 87 article-title: A short 2 week dose titration regimen reduces the severity of flu‐like symptoms with initial interferon gamma‐1b treatment publication-title: Curr Med Res Opin – volume: 42 start-page: 496 year: 2011 end-page: 505 article-title: Prolonged treatment with pimelico‐aminobenzamide HDAC inhibitors ameliorates the disease phenotype of a Friedreich ataxia mouse model publication-title: Neurobiol Dis – volume: 66 start-page: 1711 year: 2006 end-page: 6 article-title: Measuring Friedreich ataxia: complementary features of examination performance measures publication-title: Neurology – volume: 59 start-page: 743 year: 2002 end-page: 7 article-title: Friedreich ataxia: effects of genetic understanding on clinical evaluation and therapy publication-title: Arch Neurol – volume: 303 start-page: 1 year: 2011 end-page: 12 article-title: Friedreich's ataxia: pathology, pathogenesis, and molecular genetics publication-title: J Neurol Sci – volume: 276 start-page: 1709 year: 1997 end-page: 12 article-title: Regulation of mitochondrial iron accumulation by Yfh1p, a putative homolog of frataxin publication-title: Science – volume: 67 start-page: 941 year: 2010 end-page: 7 article-title: A phase 3, double‐blind, placebo‐controlled trial of idebenone in Friedreich ataxia publication-title: Arch Neurol – volume: 24 start-page: 1987 year: 2004 end-page: 95 article-title: Friedreich ataxia mouse models with progressive cerebellar and sensory ataxia reveal autophagic neurodegeneration in dorsal root ganglia publication-title: J Neurosci – volume: 25 start-page: 426 year: 2010 end-page: 32 article-title: Measuring the rate of progression in Friedreich ataxia: implications for clinical trial design publication-title: Mov Disord – volume: 29 start-page: 935 year: 2014 end-page: 9 article-title: Safety and tolerability of carbamylated erythropoietin in Friedreich's ataxia publication-title: Mov Disord – volume: 66 start-page: 1711 year: 2006 ident: 10.1111/ane.12337-BIB0016|ane12337-cit-0016 article-title: Measuring Friedreich ataxia: complementary features of examination performance measures publication-title: Neurology doi: 10.1212/01.wnl.0000218155.46739.90 – volume: 303 start-page: 1 year: 2011 ident: 10.1111/ane.12337-BIB0006|ane12337-cit-0006 article-title: Friedreich's ataxia: pathology, pathogenesis, and molecular genetics publication-title: J Neurol Sci doi: 10.1016/j.jns.2011.01.010 – volume: 64 start-page: 1261 year: 2005 ident: 10.1111/ane.12337-BIB0017|ane12337-cit-0017 article-title: Cooperative ataxia group measuring Friedreich ataxia: interrater reliability of a neurologic rating scale publication-title: Neurology doi: 10.1212/01.WNL.0000156802.15466.79 – volume: 129 start-page: 32 year: 2014 ident: 10.1111/ane.12337-BIB0010|ane12337-cit-0010 article-title: Triple therapy with deferiprone, Idebenone, and riboflavin in Friedreich's ataxia - open-label trial publication-title: Acta Neurol Scand doi: 10.1111/ane.12141 – volume: 42 start-page: 496 year: 2011 ident: 10.1111/ane.12337-BIB0014|ane12337-cit-0014 article-title: Prolonged treatment with pimelico-aminobenzamide HDAC inhibitors ameliorates the disease phenotype of a Friedreich ataxia mouse model publication-title: Neurobiol Dis doi: 10.1016/j.nbd.2011.02.016 – volume: 59 start-page: 743 year: 2002 ident: 10.1111/ane.12337-BIB0002|ane12337-cit-0002 article-title: Friedreich ataxia: effects of genetic understanding on clinical evaluation and therapy publication-title: Arch Neurol doi: 10.1001/archneur.59.5.743 – volume: 21 start-page: 2855 year: 2012 ident: 10.1111/ane.12337-BIB0015|ane12337-cit-0015 article-title: Interferon gamma upregulates frataxin and corrects the functional deficits in a Friedreich ataxia model publication-title: Hum Mol Genet doi: 10.1093/hmg/dds110 – volume: 29 start-page: 935 year: 2014 ident: 10.1111/ane.12337-BIB0011|ane12337-cit-0011 article-title: Safety and tolerability of carbamylated erythropoietin in Friedreich's ataxia publication-title: Mov Disord doi: 10.1002/mds.25836 – volume: 27 start-page: 446 year: 2012 ident: 10.1111/ane.12337-BIB0012|ane12337-cit-0012 article-title: Erythropoietin in Friedreich ataxia: no effect on frataxin in a randomized controlled trial publication-title: Mov Disord doi: 10.1002/mds.24066 – volume: 30 start-page: 1179 year: 2014 ident: 10.1111/ane.12337-BIB0021|ane12337-cit-0021 article-title: A short 2 week dose titration regimen reduces the severity of flu-like symptoms with initial interferon gamma-1b treatment publication-title: Curr Med Res Opin doi: 10.1185/03007995.2014.899209 – volume: 27 start-page: 1187 year: 2012 ident: 10.1111/ane.12337-BIB0020|ane12337-cit-0020 article-title: Cross-sectional analysis of electrocardiograms in a large heterogeneous cohort of Friedreich ataxia subjects publication-title: J Child Neurol doi: 10.1177/0883073812448461 – volume: 24 start-page: 1987 year: 2004 ident: 10.1111/ane.12337-BIB0003|ane12337-cit-0003 article-title: Friedreich ataxia mouse models with progressive cerebellar and sensory ataxia reveal autophagic neurodegeneration in dorsal root ganglia publication-title: J Neurosci doi: 10.1523/JNEUROSCI.4549-03.2004 – volume: 104 start-page: 589 year: 1981 ident: 10.1111/ane.12337-BIB0001|ane12337-cit-0001 article-title: Friedreich's ataxia: a clinical and genetic study of 90 families with an analysis of early diagnostic criteria and intrafamilial clustering of clinical features publication-title: Brain doi: 10.1093/brain/104.3.589 – volume: 226 start-page: 227 year: 2012 ident: 10.1111/ane.12337-BIB0019|ane12337-cit-0019 article-title: Binaural speech processing in individuals with auditory neuropathy publication-title: Neuroscience doi: 10.1016/j.neuroscience.2012.08.054 – volume: 101 start-page: 238 year: 2010 ident: 10.1111/ane.12337-BIB0007|ane12337-cit-0007 article-title: A rapid, noninvasive immunoassay for frataxin: utility in assessment of Friedreich ataxia publication-title: Mol Genet Metab doi: 10.1016/j.ymgme.2010.07.001 – volume: 27 start-page: 1026 year: 2012 ident: 10.1111/ane.12337-BIB0009|ane12337-cit-0009 article-title: A0001 in Friedreich ataxia: biochemical characterization and effects in a clinical trial publication-title: Mov Disord doi: 10.1002/mds.25058 – volume: 276 start-page: 1709 year: 1997 ident: 10.1111/ane.12337-BIB0004|ane12337-cit-0004 article-title: Regulation of mitochondrial iron accumulation by Yfh1p, a putative homolog of frataxin publication-title: Science doi: 10.1126/science.276.5319.1709 – volume: 67 start-page: 941 year: 2010 ident: 10.1111/ane.12337-BIB0008|ane12337-cit-0008 article-title: A phase 3, double-blind, placebo-controlled trial of idebenone in Friedreich ataxia publication-title: Arch Neurol doi: 10.1001/archneurol.2010.168 – volume: 25 start-page: 426 year: 2010 ident: 10.1111/ane.12337-BIB0018|ane12337-cit-0018 article-title: Measuring the rate of progression in Friedreich ataxia: implications for clinical trial design publication-title: Mov Disord doi: 10.1002/mds.22912 – volume: 17 start-page: 215 year: 1997 ident: 10.1111/ane.12337-BIB0005|ane12337-cit-0005 article-title: Aconitase and mitochondrial iron-sulphur protein deficiency in Friedreich ataxia publication-title: Nat Genet doi: 10.1038/ng1097-215 – volume: 3 start-page: e1958 year: 2008 ident: 10.1111/ane.12337-BIB0013|ane12337-cit-0013 article-title: HDAC inhibitors correct frataxin deficiency in a Friedreich ataxia mouse model publication-title: PLoS ONE doi: 10.1371/journal.pone.0001958 |
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This is an open‐label trial of the safety of interferon gamma‐1b (IFN‐γ) and its effect on frataxin levels and neurologic measures in 12... This is an open-label trial of the safety of interferon gamma-1b (IFN-γ) and its effect on frataxin levels and neurologic measures in 12 children with... Objectives/Aims This is an open-label trial of the safety of interferon gamma-1b (IFN-[gamma]) and its effect on frataxin levels and neurologic measures in 12... Objectives/Aims This is an open-label trial of the safety of interferon gamma-1b (IFN- gamma ) and its effect on frataxin levels and neurologic measures in 12... |
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| Title | Open-label pilot study of interferon gamma-1b in Friedreich ataxia |
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