COMPOSITIONS FOR TREATING CMT AND RELATED DISORDERS
The present invention relates to compositions and methods for the treatment of the Charcot-Marie-Tooth disease and related disorders.
1 - 13 . (canceled)
14 . A composition comprising baclofen, sorbitol and a compound selected from pilocarpine, methimazole, mifepristone, naltrexone, rapamycin, flurbiprofen, ketoprofen or salts, enantiomers, racemates or prodrugs of pilocarpin, methimazole, mifepristone, naltrexone, rapamycin, flurbiprofen or ketoprofen, for simultaneous, separate or sequential administration to a mammalian subject.
15 . The composition according to claim 14 , said composition comprising naltrexone, baclofen and D-sorbitol.
16 . The composition according to claim 14 , said composition, further comprising a pharmaceutically suitable excipient or carrier.
17 . The composition according to claim 14 , wherein said compounds are formulated with a drug eluting polymer, a biomolecule, a micelle or liposome-forming lipids or oil in water emulsions, or pegylated or solid nanoparticles or microparticles for oral or parenteral or intrathecal administration.
18 . A composition comprising:
(a) rapamycin,
(b) mifepristone or naltrexone, and
(c) a compound selected from pilocarpin, methimazole and baclofen,
for simultaneous, separate or sequential administration to a mammalian subject.
19 . The composition according to claim 18 , said composition comprising:
(a) rapamycin,
(b) mifepristone, and
(c) a compound selected from pilocarpin, methimazole or baclofen,
for simultaneous, separate or sequential administration to a mammalian subject.
20 . The composition according to claim 18 , said composition comprising rapamycin, mifepristone and pilocarpin.
21 . The composition according to claim 18 , said composition comprising rapamycin, mifepristone and baclofen.
22 . The composition according to claim 18 , said composition comprising rapamycin, mifepristone and methimazole.
23 . The composition according to claim 18 , said composition comprising rapamycin, naltrexone and methimazole.
24 . The composition according to claim 18 , said composition, further comprising a pharmaceutically suitable excipient or carrier.
25 . The composition according to claim 18 , wherein said compounds are formulated with a drug eluting polymer, a biomolecule, a micelle or liposome-forming lipids or oil in water emulsions, or pegylated or solid nanoparticles or microparticles for oral or parenteral or intrathecal administration.
26 . A method of treating Charcot-Marie-Tooth (CMT) disease or a related disorder comprising the administration of an effective amount of a composition according to claim 14 to a subject having CMT or a related disorder.
27 . The method according to claim 26 , wherein said related disorder is toxic neuropathy or amyotrophic lateral sclerosis (ALS).
28 . A method of treating Charcot-Marie-Tooth (CMT) disease or a related disorder comprising the administration of an effective amount of a composition according to claim 16 to a subject having CMT or a related disorder.
29 . The method according to claim 28 , wherein said related disorder is toxic neuropathy or amyotrophic lateral sclerosis (ALS).