Early treatment of cmt disease
AssigneePharnext
InventorsDaniel Cohen, Ilya Chumakov, Rodolphe Hajj, Aude Milet, Serguei Nabirochkin, Klaus-Armin Nave, Thomas Prukop, Michael Sereda
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Abstract
The present invention provides methods for treating, preventing, or delaying the onset of Charcot-Marie-Tooth Disease type 1A (CMT1A), by administering to a subject in need thereof a combination comprising baclofen, sorbitol and naltrexone, or a pharmaceutically acceptable salt thereof.
Claims31
1-20. (canceled)
A method of treating Charcot Marie Tooth type 1A disease (CMT1A) in a subject bearing a duplication or mutation of a PMP22 gene, comprising the step of administering to the subject a combination of baclofen, sorbitol, and naltrexone, or a pharmaceutically acceptable salt thereof, effective to treat CMT1A;
- wherein the subject is a human at an early stage of the disease and showing no or few physiological or morphological signs of CMT1A.
Dependencies are inferred from the claim text.