	US 12,433,862 B2
	Pharmaceutical compositions and uses directed to lysosomal storage disorders
Michael Strupp, Munich (DE)
Assigned to INTRABIO LIMITED, London (GB)
Filed by INTRABIO LIMITED, London (GB)
Filed on Jun. 30, 2022, as Appl. No. 17/854,027.
Application 17/854,027 is a continuation of application No. 16/324,301, granted, now 11,400,067, previously published as PCT/IB2017/054928, filed on Aug. 11, 2017.
Claims priority of application No. 1613828 (GB), filed on Aug. 11, 2016; application No. 1702552 (GB), filed on Feb. 16, 2017; application No. 1705762 (GB), filed on Apr. 10, 2017; and application No. 1706854 (GB), filed on Apr. 28, 2017.
Prior Publication US 2022/0331278 A1, Oct. 20, 2022
This patent is subject to a terminal disclaimer.
Int. Cl. A61K 31/198 (2006.01); A61K 31/13 (2006.01); A61P 25/00 (2006.01)

CPC A61K 31/198 (2013.01) [A61K 31/13 (2013.01); A61P 25/00 (2018.01)]

20 Claims

1. A method of treating a lysosomal storage disorder (LSD) or one or more symptoms associated with the LSD in a subject in need thereof comprising:

administering about 250 mg to about 15 g per day of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration of greater than 3 months,

wherein the LSD is chosen from Niemann-Pick type C disease, Tay-Sachs disease, the AB variant of Tay-Sachs disease, Sandhoff disease, Niemann-Pick type A disease, Niemann-Pick type B disease, Fabry disease, neuronal ceroid lipofuscinoses, Krabbe disease, Farber disease, Gaucher disease, metachromatic leukodystrophy, multiple sulphatase deficiency, mucolipidosis II, mucolipidosis III, MPS III, MPS VII, GM1 gangliosidosis, and aspartylglucosaminuria.