Edison Pharmaceuticals initiates Phase 2B/3 study of EPI-743 in children with...
Edison Pharmaceuticals today announced the initiation of a phase 2B/3 study entitled, "A Phase 2B/3 Open-label Study of EPI-743 in Children with Leigh Syndrome" to be conducted in conjunction with...
View ArticleFDA grants Fast Track designation to Edison’s lead drug EPI-743
Edison Pharmaceuticals today announced that the United States Food and Drug Administration granted Fast Track designation to EPI-743, the company's lead drug, for the treatment of Friedreich's ataxia....
View ArticleEdison Pharmaceuticals begins EPI-743 clinical trial in children with Pearson...
Edison Pharmaceuticals today announced the initiation of a phase 2 study entitled "A Phase 2 Safety and Efficacy Study of EPI-743 (Vincerinone™) in Children with Pearson Syndrome." The Investigative...
View ArticleTransgenomic to provide genetic testing services for Raptor’s clinical trial
Transgenomic, Inc., a global biotechnology company advancing personalized medicine in cardiology, oncology, and inherited diseases through diagnostic tests as well as clinical and research services,...
View ArticleEdison Pharmaceuticals' EPI-743 granted orphan designation for Leigh syndrome...
Edison Pharmaceuticals has announced that the Japanese Ministry of Health, Labor and Welfare (MHLW) has granted Orphan Designation to EPI-743 for the treatment of Leigh syndrome.
View ArticleEdison's EPI-743 receives FDA orphan drug designation for treatment of Leigh...
Edison Pharmaceuticals today announced that the FDA has granted Orphan Drug status to vatiquinone for the treatment of Leigh syndrome.
View ArticleSalk researchers move one step closer to making cures for genetic diseases a...
Healthy brain, muscle, eye and heart cells would improve the lives of tens of thousands of people around the world with debilitating mitochondrial diseases. Now, researchers at the Salk Institute have...
View ArticleRapamycin drug could target neural damage linked to Leigh syndrome
Salk Institute scientists showed how an FDA-approved drug boosts the health of brain cells by limiting their energy use. Like removing unnecessary lighting from a financially strapped household to save...
View ArticleScientists identify timing of major metabolic shift in developing neurons
Our brains can survive only for a few minutes without oxygen. Salk Institute researchers have now identified the timing of a dramatic metabolic shift in developing neurons, which makes them become...
View ArticlePioneering IVF technique offers hope to families with inheritable...
Details of a pioneering IVF technique using mitochondrial replacement therapy (MRT) have been revealed, giving hope to those families with inheritable mitochondrial disorders that they may be able to...
View ArticleStudy reveals how devastating mitochondrial diseases strike families without...
Researchers have shown for the first time how children can inherit a severe - potentially fatal - mitochondrial disease from a healthy mother.
View ArticleResearchers highlight the need to reconsider mitochondrial replacement...
Mothers with mitochondrial DNA mutations often give birth to children who face incurable and fatal illnesses. But a much-studied form of mitochondrial replacement could prevent the transmission of such...
View ArticleResearch team identifies Ashkenazi Jewish founder mutation for Leigh syndrome
Over 30 years ago, Marsha and Allen Barnett lost their sons to a puzzling childhood disease that relentlessly attacked their nervous systems and sapped their energy.
View ArticleStudy: Mice lacking PARL protein display specific problems in the nervous...
Leuven researchers led by professor Bart De Strooper (VIB-KU Leuven) have identified a new role for PARL, a protein that has been linked to Parkinson's disease.
View ArticleGene behind mitochondrial disease has more varied effects than previously...
For more than two decades, mutations in a gene located in the DNA of mitochondria have been classified as a mitochondrial disease and linked to a particular set of symptoms.
View ArticleResearchers identify two groups of neurons related to Leigh Syndrome
Leigh Syndrome affects one in every 40,000 newborns and is produced by deficits in the mitochondria, which are in charge of providing the energy needed for cells to function correctly.
View ArticleStudy paves the way to a potential new treatment for rare terminal childhood...
Scientists at the University of Exeter have identified a way to "rescue" cells that have genetically mutated, paving the way to a possible new treatment for rare terminal childhood illness such as...
View ArticleCombination of therapies may benefit patients with mitochondrial respiratory...
Researchers from the Mitochondrial Medicine Frontier Program at Children's Hospital of Philadelphia (CHOP) have demonstrated how one combination of therapies may be beneficial for patients with...
View ArticleResearchers develop first human model for studying the neuronal pathology of...
Leigh syndrome is the most severe mitochondrial disease in children. It causes severe muscle weakness, movement defects, and intellectual disabilities.
View ArticleResearchers identify mechanism that may lead to new therapies for strokes,...
In a surprising discovery, researchers at Massachusetts General Hospital (MGH) identified a mechanism that protects the brain from the effects of hypoxia, a potentially lethal deprivation of oxygen.
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