The United States of America, As Represented by the Secretary, Department of Health and Human Services

University of Kansas

Northwestern University

Icahn School of Medicine at Mount Sinai

Samarjit Patnaik

Juan Jose Marugan

Wei Ping Zheng

Noel Terrence Southall

Frank John Schoenen

Wendy Westbroek

Ehud Goldin

Ellen Sidransky

Kevin Frankowski

Sui Huang

Omid Motabar

Chen Wang

John Thomas Norton

Steven Titus

Steven Andrew Rogers

Marc Ferrer

Yiannis A Ioannou

Patricia Dranchak

Erika Elaine Englund

Marc Ferrer-Alegre

Seameen Dehdasthi

Hee-Yong Kim

Elma Aflaki

Udo Rudloff

Fannie Chen

Raul Rolando Calvo

Mercedes Taylor

Yuchi Chen

Altea Rocchi

Congcong He

Serguei Kozlov

John C Braisted

Wei Zheng

Wendy Westbrook

M Ehud Goldin

The invention provides a compound of formula (I): wherein R, R, R, and Rare as defined herein, ginsenoside Rg2 of structure (II): or a combination thereof, for use in treating or preventing a condition responsive to the induction of autophagy in a brain of a mammal in need thereof. 

Autophagy inducers for treatment of CNS conditions

Substituted pyrazolopyrimidines and dihydropyrazolopyrimidines and related compounds, their methods of manufacture, compositions containing these compounds, and methods of use of these compounds in treating lysosomal storage disorders such as Gaucher disease are described herein. The compounds are of general Formula (I) in which variables R-Rand X are described in the application.

Substituted pyrazolopyrimidines as glucocerebrosidase activators

The disclosure provides compounds for reducing the prevalence of the perinucleolar compartment in cells, for example, of formula (I), wherein R, R, R, and Rare as defined herein, that are useful in treating a disease or disorder associated with increased prevalence of the perinucleolar compartment, such as cancer. Also disclosed is a composition containing a pharmaceutically acceptable carrier and at least one compound embodying the principles of the invention, and a method of treating or preventing cancer in a mammal. 

Compounds and methods for the prevention and treatment of tumor metastasis and tumorigenesis

Methods and compositions for treating lysosomal storage disorders are disclosed. The methods involve administering a genus of benzenesulfonamides, particularly N-[3-(aminosulfonyl)phenyl]-benzamides and heteroarylamides. A genus of suitable compounds is shown in formula 1: 

Benzenesulfonamide upregulators of NPC1 for Neimann-Pick disease and other lysosomal storage disorders

Compounds of Formula (I) and the pharmaceutically acceptable salts thereof are disclosed. The variables. R-R, m, n, o, and p are disclosed herein. The compounds are useful for treating Gaucher disease and inhibiting the onset of Gaucher disease symptoms in a patient having a GBA gene mutation and for treating Parkinson's disease. Pharmaceutical compositions containing compounds of Formula (I) and methods of treatment comprising administering compounds of Formula (I) are also disclosed. 

Salicylic acid derivatives useful as glucocerebrosidase activators

The invention provides derivatives of DEA which have increased potency and hydrolysis resistance as compared to DEA, and compositions thereof, as well as methods of using these derivatives to promote neurogenesis, neurite growth and/or length, and/or promote synaptogenesis.

Derivatives of docosahexaenoylethanolamide and uses thereof

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Gaithersburg, MD, United States of America

Samarjit Patnaik