Redox-active therapeutics for treatment of mitochondrial diseases and other conditions and modulation of energy biomarkers
AssigneePTC Therapeutics, Inc.
InventorsGuy M. Miller, Sidney M. Hecht
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Abstract
Methods of treating or suppressing mitochondrial diseases, such as Friedreich's ataxia (FRDA), Leber's Hereditary Optic Neuropathy (LHON), mitochondrial myopathy, encephalopathy, lactacidosis, stroke (MELAS), or Kearns-Sayre Syndrome (KSS) are disclosed, as well as compounds useful in the methods of the invention, such as alpha-tocopherol quinone. Methods and compounds useful in treating other disorders are also disclosed. Energy biomarkers useful in assessing the metabolic state of a subject and the efficacy of treatment are also disclosed. Methods of modulating, normalizing, or enhancing energy biomarkers, as well as compounds useful for such methods, are also disclosed.
Claims85
A method of treating a mitochondrial disorder, comprising administering to a subject in need thereof a composition comprising a therapeutically effective amount of one or more compounds selected from the group consisting of the following formulas:
- and all stereoisomers, or mixtures of stereoisomers thereof, wherein the one or more compounds are the sole active pharmaceutical agent or agents present in a therapeutically effective amount in the composition, and
- wherein the mitochondrial disorder is selected from the group consisting of an inherited mitochondrial disease;
- Myoclonic Epilepsy with Ragged Red Fibers (MERRF);
- Mitochondrial Myopathy, Encephalopathy, Lactacidosis, Stroke (MELAS);
- Leber's Hereditary Optic Neuropathy (LHON);
- Leigh Syndrome;
- Kearns-Sayre Syndrome (KSS);
- and Friedreich's Ataxia (FA).
A method of treating epilepsy caused by a mitochondrial disorder, comprising administering to a subject in need thereof a composition comprising a therapeutically effective amount of one or more compounds selected from the group consisting of the following formulas:
- and all stereoisomers, or mixtures of stereoisomers thereof, wherein the one or more compounds are the sole active pharmaceutical agent or agents present in a therapeutically effective amount in the composition.
A method of treating a mitochondrial disorder, comprising administering to a subject in need thereof a therapeutically effective amount of one or more compounds selected from the group consisting of the following formulas:
- and all stereoisomers, or mixtures of stereoisomers thereof, and wherein the mitochondrial disorder is selected from the group consisting of an inherited mitochondrial disease;
- Myoclonic Epilepsy with Ragged Red Fibers (MERRF);
- Mitochondrial Myopathy, Encephalopathy, Lactacidosis, Stroke (MELAS);
- Leber's Hereditary Optic Neuropathy (LHON);
- Leigh Syndrome;
- Kearns-Sayre Syndrome (KSS);
- and Friedreich's Ataxia (FA).
A method treating a mitochondrial disorder, comprising administering to a subject in need thereof a composition comprising a therapeutically effective amount of one or more compounds selected from the group consisting of the following formulas:
- and all stereoisomers, or mixtures of stereoisomers thereof, wherein the one or more compounds are the sole active pharmaceutical agent or agents used to treat the disorder;
- and
- wherein the mitochondrial disorder is selected from the group consisting of an inherited mitochondrial disease;
- Myoclonic Epilepsy with Ragged Red Fibers (MERRF);
- Mitochondrial Myopathy, Encephalopathy, Lactacidosis, Stroke (MELAS);
- Leber's Hereditary Optic Neuropathy (LHON);
- Leigh Syndrome;
- Kearns-Sayre Syndrome (KSS);
- and Friedreich's Ataxia (FA).
A method of treating epilepsy caused by a mitochondrial disorder, comprising administering to a subject in need thereof a composition comprising a therapeutically effective amount of one or more compounds selected from the group consisting of the following formulas:
- and all stereoisomers, or mixtures of stereoisomers thereof.
A method of treating epilepsy caused by a mitochondrial disorder, comprising administering to a subject in need thereof a composition comprising a therapeutically effective amount of one or more compounds selected from the group consisting of the following formulas:
- and all stereoisomers, or mixtures of stereoisomers thereof, wherein the one or more compounds are the sole active pharmaceutical agent or agents used to treat the disorder.
Dependencies are inferred from the claim text.