ARIMOCLOMOL FOR TREATING GLUCOCEREBROSIDASE ASSOCIATED DISORDERS

1 . A method of increasing glucocerebrosidase (GBA) level and/or activity in a cell, comprising:

contacting the cell with an active pharmaceutical ingredient selected from: N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride, a stereoisomer thereof, and an acid addition salt thereof.

2 . The method of claim 1 , wherein the cell has reduced levels and/or activity of GBA compared to a control.

3 . The method of claim 2 , wherein the cell has one or more GBA gene mutations.

4 . The method of claim 3 , wherein the GBA gene mutation is homozygous.

5 . The method of claim 3 , wherein the GBA gene mutation is heterozygous.

6 . The method of claim 3 , wherein the GBA gene mutation is compound heterozygous.

7 . The method of claim 3 , wherein the GBA gene mutation is selected from the group consisting of L444P, D409H, D409V, E235A, E340A, E326K, N370S, N370S/1-BP ins 84G, V394L, A456P, V460V, C342G, G325R, P415R, Y133*, F213I, N188S, and IVS2+1G>A/N188S.

8 . The method of claim 2 , wherein the reduced levels and/or activity of GBA is idiopathic.

9 . The method of claim 1 , wherein the active pharmaceutical ingredient:

i) is a racemate of N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride; or

ii) is an optically active stereoisomer of N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride; or

iii) is an enantiomer of N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride; or

iv) is selected from the group consisting of (+)-R—N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride, and (−)-(S)-N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride; or

v) is an acid addition salt of N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride; or

vi) is selected from the group consisting of N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride citrate, and N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride maleate; or

vii) is selected from the group consisting of (+)-R—N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride citrate; (−)-S—N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride citrate; (+)-R—N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride maleate; and (−)-S—N-[2-hydroxy-3-(1-piperidinyl)-propoxy]-pyridine-1-oxide-3-carboximidoyl chloride maleate.

10 . The method of claim 1 , wherein the cell is in vitro or in vivo.

11 . The method of claim 1 , wherein the GBA level and/or activity is increased at least 1.5-fold.

12 . The method of claim 1 , wherein the GBA level and/or activity is increased at least 10%.