	US 12,433,863 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 Feb. 2, 2024, as Appl. No. 18/430,858.
Application 18/430,858 is a continuation of application No. 17/854,027, filed on Jun. 30, 2022.
Application 17/854,027 is a continuation of application No. 16/324,301, granted, now 11,400,067, issued on Aug. 2, 2022, 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 2024/0197663 A1, Jun. 20, 2024
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)]

27 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 a therapeutically effective amount of acetyl-leucine or a pharmaceutically acceptable salt thereof to the subject for a duration of at least about 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, and

wherein the subject achieves an improved Scale for the Assessment and Rating of Ataxia (SARA) assessment compared to before administration.