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https://www.readbyqxmd.com/read/29210930/leber-hereditary-optic-neuropathy-caused-by-a-mitochondrial-dna-10663t-c-point-mutation-and-its-response-to-idebenone-treatment
#1
Øystein Kalsnes Jørstad, Eva Meling Ødegaard, Ketil Riddervold Heimdal, Emilia Kerty, Ketil Riddervold Heimdal, Emilia Kerty
No abstract text is available yet for this article.
November 21, 2017: Journal of Neuro-ophthalmology: the Official Journal of the North American Neuro-Ophthalmology Society
https://www.readbyqxmd.com/read/29133631/mitochondrial-genetics-and-therapeutic-overview-of-leber-s-hereditary-optic-neuropathy
#2
REVIEW
Agaath Hedina Manickam, Minu Jenifer Michael, Sivasamy Ramasamy
Leber's hereditary optic neuropathy (LHON) is a common inherited mitochondrial disorder that is characterized by the degeneration of the optic nerves, leading to vision loss. The major mutations in the mitochondrial genes ND1, ND4, and ND6 of LHON subjects are found to increase the oxidative stress experienced by the optic nerve cell, thereby leading to nerve cell damage. Accurate treatments are not available and drugs that are commercially available like Idebenone, EPI-743, and Bendavia with their antioxidant role help in reducing the oxidative stress experienced by the cell thereby preventing the progression of the disease...
November 2017: Indian Journal of Ophthalmology
https://www.readbyqxmd.com/read/29054473/mitochondrial-disorders-of-the-retinal-ganglion-cells-and-the-optic-nerve
#3
REVIEW
Josef Finsterer, Michelangelo Mancuso, Davide Pareyson, Jean-Marc Burgunder, Thomas Klopstock
OBJECTIVES: To summarise and discuss recent findings and future perspectives concerning mitochondrial disorders (MIDs) affecting the retinal ganglion cells and the optic nerve (mitochondrial optic neuropathy. MON). METHOD: Literature review. RESULTS: MON in MIDs is more frequent than usually anticipated. MON may occur in specific as well as non-specific MIDs. In specific and non-specific MIDs, MON may be a prominent or non-prominent phenotypic feature and due to mutations in genes located either in the mitochondrial DNA (mtDNA) or the nuclear DNA (nDNA)...
October 18, 2017: Mitochondrion
https://www.readbyqxmd.com/read/29047345/visual-prognosis-better-in-eyes-with-less-severe-reduction-of-visual-acuity-one-year-after-onset-of-leber-hereditary-optic-neuropathy-caused-by-the-11-778-mutation
#4
Yukihiko Mashima, Kazuteru Kigasawa, Kei Shinoda, Masato Wakakura, Yoshihisa Oguchi
BACKGROUND: Patients with Leber hereditary optic neuropathy (LHON) have a progressive decrease of their visual acuity which can deteriorate to <0.1. Some patients can have a partial recovery of their vision in one or both eyes. One prognostic factor associated with a recovery of vision is an early-age onset. The purpose of this study was to determine other clinical factors that are predictive of a good visual recovery. METHODS: Sixty-one Japanese LHON patients, with the 11,778 mutation and a mean age of 23...
October 18, 2017: BMC Ophthalmology
https://www.readbyqxmd.com/read/28991104/international-consensus-statement-on-the-clinical-and-therapeutic-management-of-leber-hereditary-optic-neuropathy
#5
Valerio Carelli, Michele Carbonelli, Irenaeus F de Coo, Aki Kawasaki, Thomas Klopstock, Wolf A Lagrèze, Chiara La Morgia, Nancy J Newman, Christophe Orssaud, Jan Willem R Pott, Alfredo A Sadun, Judith van Everdingen, Catherine Vignal-Clermont, Marcela Votruba, Patrick Yu-Wai-Man, Piero Barboni
Leber hereditary optic neuropathy (LHON) is currently estimated as the most frequent mitochondrial disease (1 in 27,000-45,000). Its molecular pathogenesis and natural history is now fairly well understood. LHON also is the first mitochondrial disease for which a treatment has been approved (idebenone-Raxone, Santhera Pharmaceuticals) by the European Medicine Agency, under exceptional circumstances because of the rarity and severity of the disease. However, what remains unclear includes the optimal target population, timing, dose, and frequency of administration of idebenone in LHON due to lack of accepted definitions, criteria, and general guidelines for the clinical management of LHON...
December 2017: Journal of Neuro-ophthalmology: the Official Journal of the North American Neuro-Ophthalmology Society
https://www.readbyqxmd.com/read/28943110/therapies-for-mitochondrial-diseases-and-current-clinical-trials
#6
REVIEW
Ayman W El-Hattab, Ana Maria Zarante, Mohammed Almannai, Fernando Scaglia
Mitochondrial diseases are a clinically and genetically heterogeneous group of disorders that result from dysfunction of the mitochondrial oxidative phosphorylation due to molecular defects in genes encoding mitochondrial proteins. Despite the advances in molecular and biochemical methodologies leading to better understanding of the etiology and mechanism of these diseases, there are still no satisfactory therapies available for mitochondrial disorders. Treatment for mitochondrial diseases remains largely symptomatic and does not significantly alter the course of the disease...
September 18, 2017: Molecular Genetics and Metabolism
https://www.readbyqxmd.com/read/28869486/efficacy-of-idebenone-to-preserve-respiratory-function-above-clinically-meaningful-thresholds-for-forced-vital-capacity-fvc-in-patients-with-duchenne-muscular-dystrophy
#7
Oscar H Mayer, Mika Leinonen, Christian Rummey, Thomas Meier, Gunnar M Buyse
BACKGROUND: Patients with DMD experience progressive restrictive respiratory disease and eventual respiratory failure. Standard of care guidelines command changes in disease management when forced vital capacity percent of predicted (FVC% p) falls below clinically relevant thresholds. The Phase 3 DELOS trial in patients with DMD demonstrated that idebenone reduces the loss of peak expiratory flow and FVC compared to placebo (Buyse GM, et al.; Lancet 2015; 385 : 1748-57). OBJECTIVE: Post-hoc analyses were conducted to assess whether treatment with idebenone could reduce the risk of patients dropping below clinically meaningful thresholds of FVC% p...
2017: Journal of Neuromuscular Diseases
https://www.readbyqxmd.com/read/28699051/-assessing-the-added-benefit-of-new-ophthalmic-drugs-which-additional-insights-can-be-extracted-from-the-early-benefit-assessment
#8
REVIEW
M Appelrath, G Glaeske
Until now six ophthalmic agents have undergone the German early benefit assessment according to § 35a Social Security Code (SGB) V in a total of eleven indications. Only one agent (ocriplasmin) was recognized by the Federal Joint Committee as having an added benefit based on submitted study data for a subpopulation (indication of a considerable added benefit, limited for 5 years) and another agent, idebenone, received an added benefit due to its orphan drug designation (proof of a not quantifiable added benefit, limited for 2 years)...
July 11, 2017: Der Ophthalmologe: Zeitschrift der Deutschen Ophthalmologischen Gesellschaft
https://www.readbyqxmd.com/read/28648635/pro-oxidant-pro-inflammatory-alterations-in-the-offspring%C3%A2-s-heart-of-mild-diabetic-rats-are-regulated-by-maternal-treatments-with-a-mitochondrial-antioxidant
#9
Romina Higa, Sabrina L Roberti, Evangelina Capobianco, Daiana Fornes, Verónica White, Alicia Jawerbaum
Maternal diabetes programs metabolic and cardiovascular diseases in the offspring. Here, we demonstrated increased pro-oxidant/pro-inflammatory markers in the heart of 2-day-old offspring of diabetic rats, previous to the induction of metabolic alterations. At a pre-pubertal stage, sex-dependent changes were evidenced in the diabetic group, as only males showed increased glycemia as well as increased concentrations of nitrated proteins, matrix metalloproteinase-9 and peroxisome proliferator activated receptor α (PPARα) in the heart...
June 23, 2017: Reproductive Toxicology
https://www.readbyqxmd.com/read/28643190/mitochondrial-biogenesis-and-neural-differentiation-of-human-ipsc-is-modulated-by-idebenone-in-a-developmental-stage-dependent-manner
#10
J Augustyniak, J Lenart, M Zychowicz, P P Stepien, L Buzanska
Idebenone, the synthetic analog of coenzyme Q10 can improve electron transport in mitochondria. Therefore, it is used in the treatment of Alzheimer's disease and other cognitive impairments. However, the mechanism of its action on neurodevelopment is still to be elucidated. Here we demonstrate that the cellular response of human induced pluripotent stem cells (hiPSC) to idebenone depends on the stage of neural differentiation. When: neural stem cells (NSC), early neural progenitors (eNP) and advanced neural progenitors (NP) have been studied a significant stimulation of mitochondrial biogenesis was observed only at the eNP stage of development...
August 2017: Biogerontology
https://www.readbyqxmd.com/read/28618992/modulating-mitophagy-in-mitochondrial-disease
#11
Eszter Dombi, Heather Mortiboys, Joanna Poulton
Mitochondrial diseases may result from mutations in the maternally-inherited mitochondrial DNA (mtDNA) or from mutations in nuclear genes encoding mitochondrial proteins. Their bi-genomic nature makes mitochondrial diseases a very heterogeneous group of disorders that can present at any age and can affect any type of tissue. The autophagic-lysosomal degradation pathway plays an important role in clearing dysfunctional and redundant mitochondria through a specific quality control mechanism termed mitophagy. Mitochondria could be targeted for autophagic degradation for a variety of reasons including basal turnover for recycling, starvation induced degradation, and degradation due to damage...
June 16, 2017: Current Medicinal Chemistry
https://www.readbyqxmd.com/read/28593024/cardiovascular-mitochondrial-dysfunction-induced-by-cocaine-biomarkers-and-possible-beneficial-effects-of-modulators-of-oxidative-stress
#12
REVIEW
Manuela Graziani, Paolo Sarti, Marzia Arese, Maria Chiara Magnifico, Aldo Badiani, Luciano Saso
Cocaine abuse has long been known to cause morbidity and mortality due to its cardiovascular toxic effects. The pathogenesis of the cardiovascular toxicity of cocaine use has been largely reviewed, and the most recent data indicate a fundamental role of oxidative stress in cocaine-induced cardiovascular toxicity, indicating that mitochondrial dysfunction is involved in the mechanisms of oxidative stress. The comprehension of the mechanisms involving mitochondrial dysfunction could help in selecting the most appropriate mitochondria injury biological marker, such as superoxide dismutase-2 activity and glutathionylated hemoglobin...
2017: Oxidative Medicine and Cellular Longevity
https://www.readbyqxmd.com/read/28555014/in-vitro-antioxidant-activity-of-idebenone-derivative-loaded-solid-lipid-nanoparticles
#13
Lucia Montenegro, Maria N Modica, Loredana Salerno, Anna Maria Panico, Lucia Crascì, Giovanni Puglisi, Giuseppe Romeo
Idebenone (IDE) has been proposed for the treatment of neurodegenerative diseases involving mitochondria dysfunctions. Unfortunately, to date, IDE therapeutic treatments have not been as successful as expected. To improve IDE efficacy, in this work we describe a two-step approach: (1) synthesis of IDE ester derivatives by covalent linking IDE to other two antioxidants, trolox (IDETRL) and lipoic acid (IDELIP), to obtain a synergic effect; (2) loading of IDE, IDETRL, or IDELIP into solid lipid nanoparticles (SLN) to improve IDE and its esters' water solubility while increasing and prolonging their antioxidant activity...
May 27, 2017: Molecules: a Journal of Synthetic Chemistry and Natural Product Chemistry
https://www.readbyqxmd.com/read/28093355/evaluating-the-therapeutic-potential-of-idebenone-and-related-quinone-analogues-in-leber-hereditary-optic-neuropathy
#14
Patrick Yu-Wai-Man, Devorah Soiferman, David G Moore, Florence Burté, Ann Saada
Leber hereditary optic neuropathy (LHON) is an important cause of mitochondrial blindness among young adults. In this study, we investigated the potential of four quinone analogues (CoQ1, CoQ10, decylubiquinone and idebenone) in compensating for the deleterious effect of the m.11778G>A mitochondrial DNA mutation. The LHON fibroblast cell lines tested exhibited reduced cell growth, impaired mitochondrial bioenergetics and elevated levels of reactive oxygen species (ROS). Idebenone increased ATP production and reduced ROS levels, but the effect was partial and cell-specific...
September 2017: Mitochondrion
https://www.readbyqxmd.com/read/27940755/lysine-restriction-and-pyridoxal-phosphate-administration-in-a-nadk2-patient
#15
Frederic Tort, Olatz Ugarteburu, Maria Angeles Torres, Judit García-Villoria, Marisa Girós, Angeles Ruiz, Antonia Ribes
We report the case of a 10-year-old Spanish girl with mutations in NADK2 Prenatal central nervous system abnormalities showed ventriculomegaly, colpocephaly, and hypoplasia of the corpus callosum. At birth, axial hypotonia, uncoordinated movements, microcephaly, and generalized cerebellar atrophy were detected. Metabolic investigations revealed high lysine, lactate, and pipecolic acid levels in blood and cerebrospinal fluid. Pyruvate carboxylase and pyruvate dehydrogenase activity in fibroblasts were normal...
November 2016: Pediatrics
https://www.readbyqxmd.com/read/27881490/new-micellar-electrokinetic-chromatographic-method-for-analyzing-idebenone-in-pediatric-formulations
#16
Mario Contin, Fabián Buontempo, Cristian García Becerra, Cecilia Dobrecky, Silvia Lucangioli, Valeria Tripodi
A novel, simple and reliable method based on micellar electrokinetic chromatography with ultraviolet detection was developed to analyze idebenone in a pediatric formulation. Idebenone is a synthetic short chain benzoquinone that acts as an electron carrier in the mitochondrial electron transport chain facilitating the production of adenosine triphosphate. It can be found in two different redox states that differ in their physiological properties. Idebenone has been investigated as a treatment in several neurological disorders like Friedreich's ataxia, Leber's hereditary optic neuropathy, mitochondrial encephalomyopathies and senile dementia...
March 1, 2017: Journal of Chromatographic Science
https://www.readbyqxmd.com/read/27798429/leber-hereditary-optic-neuropathy-visual-recovery-in-a-patient-with-the-rare-m-3890g-a-point-mutation
#17
Jared J Murray, Kaitlyn W Nolan, Collin McClelland, Michael S Lee
A 15-year-old boy experienced painless vision loss in the left eye of unknown duration. Leber hereditary optic neuropathy (LHON) was suspected, despite negative testing for the 3 most common pathogenic gene mutations and idebenone 300 mg 3 times daily was prescribed. Nine months later, the patient developed right eye involvement. Complete mitochondrial genome analysis revealed 2 rare variants-m.3890G>A of the MT-ND1 gene and m.8417C>A of the MT-ATP8 gene. The former has been described in severe infantile Leigh syndrome and LHON; the latter is of unknown significance...
June 2017: Journal of Neuro-ophthalmology: the Official Journal of the North American Neuro-Ophthalmology Society
https://www.readbyqxmd.com/read/27737794/human-neuron-astrocyte-3d-co-culture-based-assay-for-evaluation-of-neuroprotective-compounds
#18
Ana Paula Terrasso, Ana Carina Silva, Augusto Filipe, Pedro Pedroso, Ana Lúcia Ferreira, Paula Marques Alves, Catarina Brito
INTRODUCTION: Central nervous system drug development has registered high attrition rates, mainly due to the lack of efficacy of drug candidates, highlighting the low reliability of the models used in early-stage drug development and the need for new in vitro human cell-based models and assays to accurately identify and validate drug candidates. 3D human cell models can include different tissue cell types and represent the spatiotemporal context of the original tissue (co-cultures), allowing the establishment of biologically-relevant cell-cell and cell-extracellular matrix interactions...
January 2017: Journal of Pharmacological and Toxicological Methods
https://www.readbyqxmd.com/read/27622560/natural-product-screening-reveals-naphthoquinone-complex-i-bypass-factors
#19
Scott B Vafai, Emily Mevers, Kathleen W Higgins, Yevgenia Fomina, Jianming Zhang, Anna Mandinova, David Newman, Stanley Y Shaw, Jon Clardy, Vamsi K Mootha
Deficiency of mitochondrial complex I is encountered in both rare and common diseases, but we have limited therapeutic options to treat this lesion to the oxidative phosphorylation system (OXPHOS). Idebenone and menadione are redox-active molecules capable of rescuing OXPHOS activity by engaging complex I-independent pathways of entry, often referred to as "complex I bypass." In the present study, we created a cellular model of complex I deficiency by using CRISPR genome editing to knock out Ndufa9 in mouse myoblasts, and utilized this cell line to develop a high-throughput screening platform for novel complex I bypass factors...
2016: PloS One
https://www.readbyqxmd.com/read/27583702/stability-study-of-oral-pediatric-idebenone-suspensions
#20
Joël Schlatter, Elodie Bourguignon, Elyes Majoul, Sofiane Kabiche, Issa-Bella Balde, Salvatore Cisternino, Jean-Eudes Fontan
Adapted forms for administration to infants are limited. The proposed study was performed to propose oral liquid formulations of idebenone in Ora-Plus and either Ora-Sweet or Ora-Sweet SF, Ora-Blend, Ora-Blend SF and Inorpha. Each formulation was stored in 30 ml amber glass bottle at 5 or 25 °C for 90 days. Idebenone contents in these suspensions, determined by a stability-indicating high-performance liquid chromatography method, remained stable at least 90 days in Inorpha when stored at the two temperatures...
March 2017: Pharmaceutical Development and Technology
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