Back to results
US 12,734,160 B2Granted
Methods for treating symptoms and disorders associated with lysosomal storage diseases
AssigneeGenzyme Corporation
InventorsNigel Patrick Somerville Crawford, Tanya Zaremba Fischer
Life of this patent
Estimating the term…
Abstract
This disclosure to methods for treating or preventing particular symptoms and disorders which are associated with lysosomal storage diseases using quinuclidine compounds of formula (I), optionally in combination with enzyme replacement therapy. This includes pain, such as abdominal pain, and dermatological disorders, such as angiokeratoma, in a patient having a disease such as Fabry disease. Also disclosed is a pharmaceutical composition comprising a quinuclidine compound for use in said methods.
Claims19
A method for treating pain associated with Fabry disease, comprising:
- administering to a human subject in need thereof an effective amount of a compound of formula (I),
- or a pharmaceutically acceptable salt or prodrug thereof, wherein:
- R1 is selected from hydrogen, halogen, cyano, nitro, hydroxy, thio, amino, C1-6-alkyl, C2-6-alkenyl, C2-6-alkynyl, C1-6-alkyloxy, C2-6-alkenyloxy, and C2-6-alkynyloxy;
- R2 and R3 are independently selected from C1-3-alkyl, or R2 and R3 together form a cyclopropyl or cyclobutyl group;
- R4, R5 and R6 are each independently selected from hydrogen, halogen, nitro, hydroxy, thio, amino, C1-6-alkyl, and C1-6-alkyloxy;
- and
- A is a 5- or 6-membered aryl or heteroaryl group.
4 more elements
Dependencies are inferred from the claim text.