Balance Therapeutics, Inc.

University of Florida Research Foundation Inc

Emory University

Lyndon Lien

Andrew Jenkins

Maurice Scott Swanson

David B Rye

Gary J Bassell

Eric Tzy-Shi Wang

Disclosed are methods of treating a disorder or disease associated with myotonic dystrophy. Methods of treating a CNS dysfunction and/or cognitive impairment associated with myotonic dystrophy in a subject comprising administering a therapeutically effective amount of a GABAA receptor antagonist or inverse agonist to the subject are disclosed. Methods of treating a myotonic dystrophy associated disease or disorder caused by mis-splicing of GABRG2 in a subject comprising administering a therapeutically effective amount of a GABAA receptor antagonist or inverse agonist to the subject are disclosed. Methods of improving cognitive function or alertness in a subject having myotonic dystrophy comprising administering a therapeutically effective amount of a GABAA receptor antagonist or inverse agonist to the subject are disclosed. Examples of the GABAA receptor antagonist or inverse agonist include flumazenil, clarithromycin, a fluoroquinolone, picrotoxin, bicuculline, gabazine, cicutoxin, oenan-thotoxin, pentylenetetrazol, Ro15-4513, sarmazenil, amentoflavone, zinc, and any combination thereof.

Treatment of conditions associated with myotonic dystrophy

Provided herein are methods, formulations and dosing regimens for treating hypersomnia in a subject. For instance, methods provided herein comprise administering a GABAchloride channel blocker. In certain embodiments, the GABAchloride channel blocker is pentylenetetrazol (PTZ).

Methods for treating hypersomnia

Provided are compounds having formula I: wherein R, R, R, R, R, R, R, R, R, and Rare as disclosed herein, or a pharmaceutically salt thereof. Pharmaceutical compositions comprising the compounds and methods of their use, for instance in treating senility, senile confusion, psychoses, psychoneuroses when anxiety and nervous tension were present, cerebral arteriosclerosis, nausea, depression, fatigue, debilitation, mild behavioral disorders, irritability, emotional instability, antisocial attitudes, anxiety, vertigo or incontinence, or symptom thereof, or in improving cognitive function in individuals, for instance, in individuals with Down syndrome and other conditions, are also provided.

Deuterated pentylenetetrazoles for improving cognitive function

Substituted pentylenetetrazoles as GABA receptor activity modulators

Growing community of inventors

Hillsborough, CA, United States of America

Lyndon Lien