Regimens for treating and preventing lysosomal disorders and degenerative disorders of the central nervous system
Described herein are dosing regimens and kits for the treatment and/or prevention of lysosomal storage disorders such as Gaucher disease. Also described are dosing regimens and kits for the treatment and/or prevention of degenerative disorders of the central nervous system, such as the synucleinopathies Parkinson's disease or Lewy Body Dementia.
1. A method for treating a patient having low β-glucocerebrosidase (GCase) activity, the method comprising:
administering to the patient an effective amount of (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or a salt thereof to enhance the GCase activity in the patient,
wherein the effective amount is in the range of about 0.75 mg/kg to about 2.5 mg/kg free base equivalent (FBE) per day and the effective amount is administered less than or equal to 7 days a week.
2. The method of claim 1 , wherein the effective amount is in the range of about 0.75 mg/kg to about 1.5 mg/kg FBE per day.
3. The method of claim 1 , wherein the (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or salt thereof is administered 7 days a week.
4. The method of claim 1 , wherein the (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or salt thereof is administered orally.
5. The method of claim 1 , wherein the salt is (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol fumarate.
6. The method of claim 1 , wherein the (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or salt thereof is administered about every 24 hours.
7. The method of claim 1 , wherein the effective amount is about 1 mg/kg FBE per day.
8. The method of claim 1 , wherein the effective amount is about 0.75 mg/kg to about 1 mg/kg FBE per day.
9. The method of claim 1 , wherein the patient is diagnosed with Gaucher disease.
10. The method of claim 1 , wherein the patient is diagnosed with Parkinson's disease.
11. A method for reducing glucosylsphingosine (GlcSph) in a patient having low β-glucocerebrosidase (GCase) activity, the method comprising:
administering to the patient an effective amount of (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or a salt thereof,
wherein the effective amount is in the range of about 0.75 mg/kg to about 2.5 mg/kg free base equivalent (FBE) per day and the effective amount is administered less than or equal to 7 days a week.
12. The method of claim 11 , wherein the salt is (3R,4R,5S)-5-(difluoromethyl)piperidine-3,4-diol fumarate.
13. The method of claim 11 , wherein the (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or salt thereof is administered orally.
14. The method of claim 11 , wherein the effective amount is about 1 mg/kg FBE per day.
15. The method of claim 11 , wherein the effective amount is in the range of about 0.75 to about 1.5 mg/kg FBE per day.
16. The method of claim 11 , wherein the effective amount is about 0.75 mg/kg to about 1 mg/kg FBE per day.
17. The method of claim 11 , wherein the (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or salt thereof is administered 7 days a week.
18. The method of claim 11 , wherein the (3R,4R,5S)-5-(difluoromethyl) piperidine-3,4-diol or salt thereof is administered about every 24 hours.