US 12,226,413 B2
Early treatment of CMT disease
Daniel Cohen, Saint Cloud (FR); Ilya Chumakov, Vaux le Pénil (FR); Rodolphe Hajj, St. Germain en Laye (FR); Aude Milet, Antony (FR); Serguei Nabirochkin, Chatenay Malabry (FR); Klaus-Armin Nave, Göttingen (DE); Thomas Prukop, Duderstadt (DE); and Michael Sereda, Göttingen (DE)
Assigned to PHARNEXT, Issy les Moulineaux (FR)
Filed by Pharnext, Paris (FR)
Filed on Mar. 8, 2021, as Appl. No. 17/194,703.
Application 17/194,703 is a continuation of application No. 16/505,000, filed on Jul. 8, 2019, granted, now 10,940,147.
Application 16/505,000 is a continuation of application No. 15/619,074, filed on Jun. 9, 2017, granted, now 10,383,870, issued on Aug. 20, 2019.
Claims priority of provisional application 62/348,276, filed on Jun. 10, 2016.
Prior Publication US 2021/0260053 A1, Aug. 26, 2021
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 31/485 (2006.01); A61K 31/047 (2006.01); A61K 31/197 (2006.01); C12Q 1/6883 (2018.01)
CPC A61K 31/485 (2013.01) [A61K 31/047 (2013.01); A61K 31/197 (2013.01); C12Q 1/6883 (2013.01); C12Q 2600/156 (2013.01)] 25 Claims
 
1. 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 of 7 years of age or less; is at an early stage of the disease; and is showing no or few physiological or morphological signs of CMT1A; and wherein the method provides in said subject a long term reduction of a sign, symptom, or clinical manifestation of CMT1A for at least 1 year.