US2021077421A1PendingUtilityA1

Treatment of syngap1 encephalopathy

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Assignee: ZYNERBA PHARMACEUTICALS INCPriority: Sep 17, 2019Filed: Sep 16, 2020Published: Mar 18, 2021
Est. expirySep 17, 2039(~13.2 yrs left)· nominal 20-yr term from priority
Inventors:Terri SebreeJohn MessenheimerDonna Gutterman
A61K 31/658A61P 25/08A61P 25/28A61K 36/00A61P 25/20A61K 9/0014A61M 2021/0077A61M 21/02A61K 31/05
69
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Claims

Abstract

The present technology relates to methods of treating SYNGAP1 encephalopathy by transdermally administering an effective amount of cannabidiol (CBD) to the subject, wherein a SYNGAP1 symptom is treated in the subject.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . A method of treating SYNGAP1 encephalopathy, the method comprising:
 transdermally administering an effective amount of cannabidiol (CBD) to the subject, wherein a SYNGAP1 symptom is treated in the subject.   
     
     
         2 . The method of  claim 1 , wherein the effective amount of CBD is 250, 500, 750, or 1000 mg total daily. 
     
     
         3 . The method of  claim 1 , wherein the effective amount of CBD is 750 mg total daily. 
     
     
         4 . The method of  claim 1 , wherein the effective amount of CBD is 1000 mg total daily. 
     
     
         5 . The method according to  claim 1 , wherein the treated SYNGAP1 symptom is one or more behavioral problems. 
     
     
         6 . The method of  claim 5 , wherein the treatment of one or more behavioral problems includes a decrease in one or more behavioral problems. 
     
     
         7 . The method of  claim 6 , wherein the one or more behavioral problems comprise social avoidance, attention deficits, impulsivity, mood abnormalities, inattention, impulsivity, or physical aggression. 
     
     
         8 . The method according to  claim 1 , wherein the treated SYNGAP1 symptom is seizures. 
     
     
         9 . The method of  claim 8 , wherein treatment includes a decrease from about 25% to about 45% in SYNGAP1 seizures. 
     
     
         10 . The method of  claim 9 , wherein the SYNGAP1 seizures are at least one of focal impaired awareness seizures (FIAS) or atonic seizures (AT). 
     
     
         11 . The method according to  claim 1 , wherein two or more SYNGAP1 symptoms are treated. 
     
     
         12 . The method of  claim 11 , wherein the two or more treated SYNGAP1 symptoms are behavioral problems and SYNGAP1 seizures. 
     
     
         13 . The method of  claim 12 , wherein the treatment of behavioral problems includes a decrease in one or more behavioral problems, and wherein the treatment of SYNGAP1 seizures includes a decrease from about 25% to about 45% in SYNGAP1 seizures. 
     
     
         14 . The method of  claim 13 , wherein the behavioral problems comprise social avoidance, attention deficits, impulsivity, mood abnormalities, inattention, impulsivity, or physical aggression. 
     
     
         15 . The method of  claim 13 , wherein the SYNGAP1 seizures are at least one of focal impaired awareness seizures (FIAS) or atonic seizures (AT). 
     
     
         16 . The method according to  claim 1 , wherein treatment includes an improvement in every day satisfaction and well-being. 
     
     
         17 . The method according to  claim 1 , wherein treatment includes an improvement in cognition and awareness. 
     
     
         18 . The method according to  claim 1 , wherein the treatment includes a meaningful monthly reduction in focal impaired awareness seizures and convulsive seizures. 
     
     
         19 . The method according to  claim 1 , wherein the CBD is administered in a single daily dose or in two daily doses. 
     
     
         20 . The method according to  claim 1 , wherein the CBD is a synthetic CBD. 
     
     
         21 . The method of  claim 20 , wherein the CBD is purified. 
     
     
         22 . The method according to  claim 1 , wherein the CBD is botanically derived.

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