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.

We claim: 
     
         1 - 11 . (canceled) 
     
     
         12 . A method for treating Charcot-Marie-Tooth (CMT) disease of type 1 A (CMT1A), comprising administering to a subject in need thereof an effective amount of a GABA-B receptor agonist or a salt thereof. 
     
     
         13 . The method of  claim 12 , wherein the GABA-B receptor agonist or a salt thereof are formulated as a controlled release formulation. 
     
     
         14 . The method of  claim 12 , wherein the GABA-B receptor agonist or a salt thereof, is administered orally. 
     
     
         15 . The method of  claim 12 , wherein the GABA-B receptor agonist, or a salt thereof, is formulated in a unit dosage form. 
     
     
         16 . The method of  claim 12 , wherein pmp22 expression in Schwann cells of the subject is reduced. 
     
     
         17 . The method of  claim 12 , wherein the strength or motor performance of the subject is improved. 
     
     
         18 . The method of  claim 12 , wherein the GABA-B receptor agonist or a salt thereof is administered orally in a unit dosage form. 
     
     
         19 . The method of  claim 12 , comprising administering to the subject an effective amount of a GABA-B receptor agonist, or a salt thereof, optionally in combination with an excipient, wherein none of the following is administered to the subject: baclofen; naltrexone; pilocarpine; methimazole; mifepristone; or ketoprofen; or a salt thereof. 
     
     
         20 . The method of  claim 19 , wherein the GABA-B receptor agonist is administered orally in a unit dosage form.