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Stealth Biotherapeutics: New Treatments for Genetic Mitochondrial Diseases
Stealth Biotherapeutics is one of a new group of pharmaceutical companies developing drug treatments for genetic mitochondrial disease and acquired mitochondrial dysfunction, including heart failure, diabetic macular edema, certain skeletal muscle disorders, and rare inherited diseases such as Leber’s Hereditary Optic Neuropathy (LHON). Treating Mitochondrial Dysfunction Mitochondrial disorders are believed to arise either as a result…
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Edison Pharmaceuticals: Dedicated to the Treatment of Mitochondrial Disease
Edison Pharmaceuticals is one of a new group of pharmaceutical companies developing drug treatments for inherited mitochondrial diseases such as Leigh syndrome, Friederich’s ataxia, Leber’s hereditary optic neuropathy (LHON), and acquired mitochondrial dysfunction in Parkinson’s disease and ALS. Inherited Mitochondrial Disease Inherited mitochondrial diseases are genetic disorders caused by defects in the cells’ ability to make…
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Raptor Pharmaceuticals’ RP103: Treatment Hope for Leigh Syndrome?
Raptor Pharmaceuticals is dedicated to developing medicines that alter cellular metabolic pathways for patients with rare, debilitating, and potentially fatal diseases, including inherited mitochondrial disorders. Raptor has two FDA-approved medications: RP103 (PROCYSBI) and MP376 (QUINSAIR). RP103 was the first brought to market for the treatment of Nephropathic Cystinosis, a rare metabolic disease. It is now being…