Therapeutic approaches for treating cmt and related disorders

Abstract

Compositions and methods for the treatment of Charcot-Marie-Tooth disease and related disorders. Also provided are combination therapies for treating this disease by decreasing PMP22 expression in a subject.

1 . A method for treating Charcot-Marie-Tooth (CMT) disease or a related disorder selected from Dejerine-Sottas syndrome (DSS) and congenital hypomyelinating neuropathy (CHN), comprising administering to a subject in need thereof an effective amount of a compound selected from D-sorbitol, baclofen, or naltrexone, or a salt or a sustained release formulation thereof. 
     
     
         2 . The method of  claim 1 , wherein D-sorbitol is administered at a daily dose of 1 to 50 g. 
     
     
         3 . The method of  claim 1 , wherein baclofen is administered at a daily dose of 2 to 20 mg. 
     
     
         4 . The method of  claim 1 , wherein naltrexone is administered at a daily dose of 1 to 20 mg. 
     
     
         5 . The method of  claim 1 , wherein said compound is the only active agent. 
     
     
         6 . The method of  claim 1 , which comprises administering to the subject D-sorbitol and naltrexone; or baclofen and naltrexone, or salts or sustained release formulations thereof. 
     
     
         7 . The method of  claim 1 , wherein said compound is administered orally. 
     
     
         8 . The method of  claim 1 , wherein said compound is formulated in unit dosage forms. 
     
     
         9 . The method of  claim 1 , for treating Charcot-Marie-Tooth disease of type 1 A (CMT1A). 
     
     
         10 . A method of  claim 1 , to reduce pmp22 expression in Schwann cells of the subject. 
     
     
         11 . A method of  claim 1 , to improve the strength or motor performance of the subject