Compositions and methods to treat Niemann-Pick disease
View Patent ↗The present disclosure is directed to compositions comprising bryostatin-1, and methods comprising administering a composition comprising bryostatin-1, to treat Niemann-Pick Type C in a subject in need thereof.
1. A composition comprising bryostatin-1 in an effective amount to treat Niemann-Pick Type C (NPC), wherein the effective amount results in a concentration of bryostatin-1 in the brain of a subject in need of treatment ranging from about 0.1 nM to about 1.5 nM.
2. The composition of claim 1 , wherein the effective amount results in a concentration of bryostatin-1 in the brain of about 1 nM.
3. A method for treating Niemann-Pick Type C (NPC) in a subject in need of treatment, comprising administering to the subject a composition comprising bryostatin-1 in an effective amount to treat NPC, wherein the effective amount results in a concentration of bryostatin-1 in the brain ranging from about 0.1 nM to about 1.5 nM.
4. The method of claim 3 , wherein the effective amount results in a concentration of bryostatin-1 in the brain of about 1 nM.