Compositions for treating CMT and related disorders

1. A pharmaceutical composition comprising:

(i) baclofen or a pharmaceutically acceptable salt thereof,

(ii) sorbitol or a pharmaceutically acceptable salt thereof, and

(iii) naltrexone or a pharmaceutically acceptable salt thereof; and

a pharmaceutically acceptable excipient or carrier;

wherein the baclofen or a pharmaceutically acceptable salt thereof, sorbitol or a pharmaceutically acceptable salt thereof, and naltrexone or a pharmaceutically acceptable salt thereof are in a relative weight ratio of about 8.6:300:1.

2. The composition according to claim 1 , wherein the sorbitol or a pharmaceutically acceptable salt thereof is D-sorbitol or a pharmaceutically acceptable salt thereof.

3. The composition according to claim 1 , wherein the composition is formulated for oral administration.

4. The composition according to claim 1 , wherein the composition is a liquid formulation.

5. The composition according to claim 1 , wherein the composition contains no additional active agents.

6. The composition according to claim 1 , wherein the baclofen or a pharmaceutically acceptable salt thereof, sorbitol or a pharmaceutically acceptable salt thereof, and naltrexone or a pharmaceutically acceptable salt thereof are present in an amount effective to treat Charcot-Marie-Tooth Disease (CMT) in a human subject.

7. The composition according to claim 6 , wherein the CMT is CMT1A.

8. The composition according to claim 1 , wherein the composition is a liquid formulated for oral administration.

9. The composition according to claim 1 , wherein the composition comprises baclofen or a pharmaceutically acceptable salt thereof in an amount between about 1 and about 300 μg/kg of a human subject.

10. The composition according to claim 1 , wherein the composition comprises baclofen or a pharmaceutically acceptable salt thereof in an amount between 10 and 200 μg/kg of a human subject.

11. The composition according to claim 1 , wherein the composition comprises naltrexone or a pharmaceutically acceptable salt thereof in an amount between about 1 and about 100 μg/kg of a human subject.

12. The composition according to claim 1 , wherein the composition comprises naltrexone or a pharmaceutically acceptable salt thereof in an amount between 1 and 50 μg/kg of a human subject.

13. The composition according to claim 1 , wherein the composition comprises baclofen or a pharmaceutically acceptable salt thereof in an amount between about 1 and about 300 μg/kg of a human subject and naltrexone or a pharmaceutically acceptable salt thereof in an amount between about 1 and about 100 μg/kg of the human subject.

14. The composition according to claim 1 , wherein the composition comprises baclofen or a pharmaceutically acceptable salt thereof in an amount between 10 and 200 μg/kg of a human subject and naltrexone or a pharmaceutically acceptable salt thereof in an amount between 1 and 50 μg/kg of the human subject.

15. A unit dosage form comprising:

(i) baclofen or a pharmaceutically acceptable salt thereof in an amount between about 1 and about 300 μg/kg of a human subject,

(ii) sorbitol or a pharmaceutically acceptable salt thereof, and

(iii) naltrexone or a pharmaceutically acceptable salt thereof in an amount between about 1 and about 100 μg/kg of the human subject; and

a pharmaceutically acceptable excipient or carrier;

wherein the baclofen or a pharmaceutically acceptable salt thereof, sorbitol or a pharmaceutically acceptable salt thereof, and naltrexone or a pharmaceutically acceptable salt thereof are in a relative weight ratio of about 8.6:300:1.

16. The unit dosage form according to claim 15 , wherein the sorbitol or a pharmaceutically acceptable salt thereof is D-sorbitol.

17. The unit dosage form according to claim 16 , wherein the baclofen or a pharmaceutically acceptable salt thereof is RS baclofen.

18. The unit dosage form according to claim 17 , wherein the unit dosage form is a liquid formulated for oral administration.

19. The unit dosage form according to claim 18 , comprising RS baclofen in an amount between 10 and 200 μg/kg of the human subject and naltrexone or a pharmaceutically acceptable salt thereof in an amount between 1 and 50 μg/kg of the human subject.

20. A patient pack comprising a liquid formulation for oral administration, comprising a composition comprising:

(i) RS baclofen or a pharmaceutically acceptable salt thereof,

(ii) D-sorbitol or a pharmaceutically acceptable salt thereof, and

(iii) naltrexone or a pharmaceutically acceptable salt thereof;

a pharmaceutically acceptable excipient or carrier; and, optionally,

instructions for administration of the liquid formulation;

wherein the RS baclofen or a pharmaceutically acceptable salt thereof, D-sorbitol or a pharmaceutically acceptable salt thereof, and naltrexone or a pharmaceutically acceptable salt thereof are in a relative weight ratio of about 8.6:300:1.

21. The patient pack according to claim 20 , wherein the liquid formulation comprises divided doses.

22. The patient pack according to claim 20 , wherein the liquid formulation comprises RS baclofen or a pharmaceutically acceptable salt thereof in an amount between about 1 and about 300 μg/kg of a human subject and naltrexone or a pharmaceutically acceptable salt thereof in an amount between about 1 and about 100 μg/kg of the human subject.

23. The patient pack according to claim 21 , wherein each of the divided doses comprises RS baclofen or a pharmaceutically acceptable salt thereof in an amount between about 1 and about 300 μg/kg of a human subject and naltrexone or a pharmaceutically acceptable salt thereof in an amount between about 1 and about 100 μg/kg of the human subject.

24. The patient pack according to claim 21 , wherein each of the divided doses comprises RS baclofen or a pharmaceutically acceptable salt thereof in an amount between 10 and 200 μg/kg of a human subject and naltrexone or a pharmaceutically acceptable salt thereof in an amount between 1 and 50 μg/kg of the human subject.