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الإعدادات

الإعدادات

1. WO2017096270 - METHODS FOR TREATING MITOCHONDRIAL DISEASES

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WHAT IS CLAIMED IS:

1. A method for preventing, alleviating, attenuating the progression of, or treating a mitochondrial disease in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of a drug selected from the group consisting of papaverine, Zolpidem, a nucleotide metabolism inhibitor, methoxamine, methenamine, pharmaceutically acceptable salts thereof, analogs thereof, and combinations thereof.

2. The method of claim 1 , wherein the nucleotide metabolism inhibitor is selected from the group consisting of methotrexate, azathioprine, fluorouracil, zidovudine, pharmaceutically acceptable salts thereof, analogs thereof, and combinations thereof.

3. The method of claim 1 , wherein the drug is administered orally, ocularly, topically, systemically, intravenously, subcutaneously, intraperitoneally, intramuscularly, trans dermally, or transmucosally.

4. The method of claim 1, wherein the therapeutically effective amount of the drug is an amount sufficient to stimulate mitochondrial ATP synthesis and/or to inhibit the induction of one or more inflammatory genes.

5. The method of claim 1 , further comprising administering a therapeutically effective amount of rapamycin, a pharmaceutically acceptable salt thereof, or an analog thereof.

6. The method of claim 5, wherein rapamycin is administered orally, ocularly, topically, systemically, intravenously, subcutaneously, intraperitoneally, intramuscularly, trans dermally, or transmucosally.

7. The method of claim 5, wherein the therapeutically effective amount of rapamycin is an amount sufficient to inhibit the induction of one or more inflammatory genes.

8. The method of claim 1 , further comprising administering a therapeutically effective amount of idebenone, a pharmaceutically acceptable salt thereof, or an analog thereof.

9. The method of claim 8, wherein the therapeutically effective amount of idebenone is an amount sufficient to stimulate mitochondrial ATP synthesis and/or to inhibit the induction of one or more inflammatory genes.

10. The method of claim 1 , wherein the mitochondrial disease leads to vision loss or blindness.

11. The method of claim 1, wherein the mitochondrial disease is selected from the group consisting of Leigh syndrome; Leber's hereditary optic neuropathy (LHON); Alpers-Huttenlocher syndrome; ataxia neuropathy syndromes (ANS); chronic progressive external opthalmoplegia (CPEO); diabetes mellitus and deafness (DAD); dominant optic atrophy (DOA); Friedreich's ataxia (FRDA); infantile myopathy and lactic acidosis; Kearns-Sayre Syndrome (KSS); myoclonic epilepsy with ragged red fibers (MERRF); mitochondrial myopathy, encephalomyopathy, lactic acidosis, stroke (MELAS); myoclonic epilespy myopathy sensory ataxia (MEMSA); mitochondrial neurogastrointestinal encephalopathy (MNGIE); neuropathy, ataxia, retinitis pigmentosa, and ptosis (NARP); Pearson syndrome; and Sengers syndrome.

12. The method of claim 1 , wherein the mitochondrial disease is Leigh syndrome or Leber's hereditary optic neuropathy (LHON).

13. The method of claim 1, wherein the subject has a likelihood of having or developing the mitochondrial disease.

14. The method of claim 1 , wherein the subject has at least one genetic mutation associated with the mitochondrial disease.

15. The method of claim 1 , wherein the subject is clinically asymptomatic.

16. The method of claim 1 , wherein the subject has at least one clinical symptom of the mitochondrial disease.

17. The method of claim 16, wherein the at least one clinical symptom comprises vision loss or blindness.

18. A method for preventing, alleviating, attenuating the progression of, or treating a mitochondrial disease in a subject in need thereof, comprising administering to the subject a therapeutically effective amount of rapamycin, a pharmaceutically acceptable salt thereof, or an analog thereof,

wherein the mitochondrial disease is selected from the group consisting of Leber's hereditary optic neuropathy (LHON); Alpers-Huttenlocher syndrome; ataxia neuropathy syndromes (ANS); chronic progressive external opthalmoplegia (CPEO); diabetes mellitus and deafness (DAD); dominant optic atrophy (DOA); Friedreich's ataxia (FRDA); infantile myopathy and lactic acidosis; Kearns-Sayre Syndrome (KSS); myoclonic epilepsy with ragged red fibers (MERRF); mitochondrial myopathy, encephalomyopathy, lactic acidosis, stroke (MELAS); myoclonic epilespy myopathy sensory ataxia (MEMSA); mitochondrial neurogastrointestinal encephalopathy (MNGIE); neuropathy, ataxia, retinitis pigmentosa, and ptosis (NARP); Pearson syndrome; and Sengers syndrome.

19. The method of claim 18, wherein the mitochondrial disease is Leber's hereditary optic neuropathy (LHON).

20. The method of claim 18, wherein rapamycin is administered orally, ocularly, topically, systemically, intravenously, subcutaneously, intraperitoneally, intramuscularly, trans dermally, or transmucosally.

21. The method of claim 18, wherein the therapeutically effective amount of rapamycin is an amount sufficient to inhibit the induction of one or more inflammatory genes.

22. The method of claim 18, further comprising administering a therapeutically effective amount of a drug selected from the group consisting of papaverine, Zolpidem, a nucleotide metabolism inhibitor, methoxamine, methenamine, idebenone, pharmaceutically acceptable salts thereof, analogs thereof, and combinations thereof.

23. The method of claim 22, wherein the nucleotide metabolism inhibitor is selected from the group consisting of methotrexate, azathioprine, fluorouracil, zidovudine, pharmaceutically acceptable salts thereof, and analogs thereof.

24. The method of claim 22, wherein the drug is administered orally, ocularly, topically, systemically, intravenously, subcutaneously, intraperitoneally, intramuscularly, trans dermally, or transmucosally.

25. The method of claim 22, wherein the therapeutically effective amount of the drug is an amount sufficient to stimulate mitochondrial ATP synthesis and/or to inhibit the induction of one or more inflammatory genes.

26. The method of claim 18, wherein the subject has a likelihood of having or developing the mitochondrial disease.

27. The method of claim 18, wherein the subject has at least one genetic mutation associated with the mitochondrial disease.

28. The method of claim 18, wherein the subject is clinically asymptomatic.

29. The method of claim 18, wherein the subject has at least one clinical symptom of the mitochondrial disease.

30. The method of claim 29, wherein the at least one clinical symptom comprises vision loss or blindness.