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

University of Kansas

Universiteit Gent

Pierre Fabre Dermo-Cosmetique

Wendy Westbroek

Wei Ping Zheng

Juan Jose Marugan

Noel Terrence Southall

Samarjit Patnaik

Ehud Goldin

Ellen Sidransky

Omid Motabar

Frank John Schoenen

Steven Andrew Rogers

Olivier De Wever

An Hendrix

Anne-Marie Schmitt-Milas

Jo Lambert

Mireille Van Gele

Elma Aflaki

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

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 present invention relates to evaluating the prognosis of patients with estrogen receptor-positive breast cancer on the basis of Rab27B expression. The invention further relates to a kit comprising an assay for measuring Rab27B levels in said patients and to the usage of Rab27B as a target to screen for drugs capable of inhibiting or diminishing metastasis of said cancer. Furthermore, the invention discloses compounds which can be used to treat estrogen receptor-positive breast cancer.

Use of the GTPase Rab27B to diagnose and treat poor prognosis estrogen-receptor-positive breast cancer

The present invention relates to novel isolated siRNAs comprising a sense RNA strand and a complementary antisense RNA strand which together form an RNA duplex, characterized in that the sense RNA strand comprises a sequence which has at most one nucleotide that is distinct in relation to a fragment of 14 to 30 contiguous nucleotides of the nucleotide sequence of exon F of the gene encoding the myosin Va protein, and also to compositions comprising at least one such siRNA, and to the use of at least one such siRNA as a cosmetic or therapeutic agent for skin depigmentation.

Anti-myosin Va siRNA and skin depigmentation

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Rockville, MD, United States of America

Wendy Westbroek