Therapeutic approaches for treating CMT and related disorders
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 of type 1 A (CMT1A), comprising administering to a subject in need thereof an effective amount of D-sorbitol, or a salt thereof or a sustained release formulation thereof.
2. The method of claim 1 , wherein D-sorbitol or a salt thereof is administered at a daily dose of about 1 to about 50 g.
3. The method of claim 1 , wherein D-sorbitol, or a salt thereof or a sustained release formulation thereof, is administered orally.
4. The method of claim 1 , wherein D-sorbitol, or a salt thereof or a sustained release formulation thereof, is formulated in a unit dosage form.
5. The method of claim 1 , wherein pmp22 expression in Schwann cells of the subject is reduced.
6. The method of claim 1 , wherein the strength or motor performance of the subject is improved.
7. The method of claim 1 , wherein D-sorbitol or a salt thereof is administered orally in a unit dosage form at a daily dose of about 1 to about 50 g.
8. The method of claim 1 , comprising administering to the subject an effective amount of D-sorbitol 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 or prodrug thereof.
9. The method of claim 8 , wherein D-sorbitol is administered orally in a unit dosage form at a daily dose of 1 to 50 g.