Leber's Hereditary Optic Neuropathy

Rudolph et al: Effects of idebenone on color vision in patients with leber hereditary optic neuropathy.J Neuroophthalmol. 2013 33:30-36

Klopstock et al: Persistence of the treatment effect of idebenone in Leber's hereditary optic neuropathy. Brain. 2013 136(Pt 2):e230

Mascialino et al: Meta-analysis of the prevalence of Leber hereditary optic neuropathy mtDNA mutations in Europe. Eur J Ophthalmol. 2012; 22:461-465

Sabet-Peyman et al: Is Leber Hereditary Optic Neuropathy Treatable? Encouraging Results With Idebenone in Both Prospective and Retrospective Trials and An Illustrative Case. J Neuro-Ophtalmol 2012; 32: 54-57

Heitz et al: Idebenone Protects against Retinal Damage and Loss of Vision in a Mouse Model of Leber's Hereditary Optic Neuropathy. PLoS ONE. 2012; 7(9): e45182

Klopstock et al: A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy. Brain. 2011; 134:2677-86

Carelli et al: Idebenone Treatment In Leber's Hereditary Optic Neuropathy. Brain. 2011; 134:e188

Sadun et al: Leber’s Hereditary Optic Neuropathy. Current Treatment Options. Neurology. 2011; 13(1):109-17

Newman: Treatment of Leber hereditary optic neuropathy. Brain. 2011; doi:10.1093/brain/ awr192
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Duchenne Muscular Dystrophy

Buyse et al: Effects of Glucocorticoids and Idebenone on Respiratory Function in Patients With Duchenne Muscular Dystrophy. Pediatric Pulmonology 2012; DOI 10.1002/ppul.22688

Buyse et al: Idebenone as a novel, therapeutic approach for Duchenne muscular dystrophy: results from a 12 month, double-blind, randomized placebo-controlled trial. Neuromuscul Disord. 2011; 21(6):396-405

Buyse et al: Long-term blinded placebo-controlled study of SNT-MC17/idebenone in the dystrophin deficient mdx mouse: cardiac protection and improved exercise performance. Eur Heart J. 2009; 30(1):116-24.

Willmann et al: Mammalian animal models for Duchenne muscular dystrophy. Neuromuscul Disord. 2009; 19(4):241-9. Review

Briguet et al: Effect of calpain and proteasome inhibition on Ca2+ dependent proteolysis and muscle histopathology in the mdx mouse. FASEB J. 2008; 22(12):4190-200

Weyermann et al: Synthesis and Evaluation of Calpain Inhibitors Carrying Muscle Cell Targeting Groups Letters in Drug Design & Discovery 2006; 3: 152-158

Courdier-Fruh et al: Utrophin Is a Calpain Substrate in Muscle Cells. Muscle & Nerve 2006; 33: 753-759

Lescop et al: Novel cell-penetrating alpha-keto-amide calpain inhibitors as potential treatment for muscular dystrophy. Bioorg Med Chem Letters 2005; 15: 5176-5181

De Luca et al: A Multidisciplinary Evaluation of the Effectiveness of Cyclosporine A in Dystrophic Mdx Mice. American Journal of Pathology 2005; 166(2):477-89

Briguet et al: Histological parameters for the quantitative assessment of muscular dystrophy in the mdx-mouse. Neuromusc. Disord. 2004; 14: 675-682

Courdier- Fuh et al: Detection of glucocorticoid-like activity in traditional Chinese medicine used for the treatment of Duchenne muscular dystrophy. Neuromusc. Disord. 2003; 13: 699-704

Briguet et al: Transcriptional activation of the utrophin promoter B by a constitutively active Ets-transcription factor. Neuromusc. Disord. 2003; 13: 143-150

Courdier-Fruh et al: Glucocorticoid-mediated regulation of utrophin levels in human muscle fibers. Neuromusc. Disord. 2002; 12: S95-S104
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Friedreich's Ataxia

Metz et al: Rating disease progression of Friedreich’s ataxia by the International Cooperative Ataxia Rating Scale: analysis of a 603-patient database. Brain. 2013;136; 259–268

Weidemann et al: The Heart in Friedreich Ataxia. Definition of Cardiomyopathy, Disease Severity, and Correlation With Neurological Symptoms. Circulation 2012; 10.1161:1626-1635

Meier et al:Assessment of neurological efficacy of idebenone in pediatric patients with Friedreich's ataxia: data from a 6-month controlled study followed by a 12-month open-label extension study. J Neurol. 2012; 259:284–291

Lagedrost et al: Idebenone in Friedreich ataxia cardiomyopathy-results from a 6-month phase III study (IONIA). Am Heart J. 2011; 161(3):639-645.e1

Lynch et al: A phase 3, double-blind, placebo-controlled trial of idebenone in friedreich ataxia. Arch Neurol. 2010; 67(8):941-7

Meier and Buyse: Idebenone: an emerging therapy for Friedreich ataxia. J Neurol. 2009; 256 Suppl 1:25-30. Review

Schulz et al: Clinical experience with high-dose idebenone in Friedreich ataxia. J Neurol. 2009; 256 Suppl 1:42-5. Review

Di Prospero NA et al: Neurological effects of high-dose idebenone in patients with Friedreich's ataxia: a randomised, placebo-controlled trial. Lancet Neurol. 2007; 6(10):878-86

Di Prospero NA et al: Safety, tolerability, and pharmacokinetics of high-dose idebenone in patients with Friedreich ataxia. Arch Neurol. 2007; 64(6):803-8

Jauslin et al: Mitochondria-targeted antioxidants protect Friedreich Ataxia fibroblasts from endogenous oxidative stress more effectively than untargeted antioxidants. The FASEB J. 2003; 17:1972-74

Jauslin et al: A cellular model for Friedreich Ataxia reveals small-molecule glutathione peroxidase mimetics as novel treatment strategy. Hum. Mol. Genet, 2002. 11:3055-3063
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Idebenone - Pharmacology and Pharmacokinetics

Erb et al: Features of Idebenone and related short-chain quinones that rescue ATP levels under conditions of impaired mitochondrial complex I. PLoS ONE. 2012; 7(4): e36153

Haefeli et al: NQO1-dependent redox cycling of Idebenone: Effects on cellular redox potential and energy levels. PLoS ONE. 2011; 6(3): e17963

Becker et al: Pharmacokinetic evaluation of idebenone. Expert Opin Drug Metab Toxicol. 2010; 6(11):1437-44

Bodmer et al: Pharmacokinetics and metabolism of idebenone in healthy male subjects. European Journal of Clinical Pharmacology: 2009; 65(5):493-501

Kutz et al: Pharmacokinetic properties and metabolism of idebenone. J Neurol. 2009; 256 Suppl 1:31-5
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Congenital Muscular Dystrophy

Meinen et al: Apoptosis inhibitors and mini-agrin have additive benefits in congenital muscular dystrophy mice. EMBO Mol Med. 2011; 3(8):465-79

Erb et al: Omigapil Ameliorates the Pathology of Muscle Dystrophy Caused by Laminin-α 2 Deficiency. The Journal of Pharmacology and Experimental Thearapeutics, 2009, Vol. 331, No. 3, JPET 331:787–795
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Dyskinesia in Parkinson's Disease

LeWitt et al: Randomized clinical trial of fipamezole for dyskinesia in Parkinson disease (FJORD study). Neurology. 2012.79:163–169

LeWitt et al: Validating Rating Performance of a New Rating Scale for Levodopa-Induced Dyskinesia (LIDS). Poster, MDS 13th International Congress of Parkinson’s Disease and Movement Disorders, 2009.