Abstract: Compounds and compositions are provided for treatment or amelioration of one or more symptoms of ?-synuclein toxicity, ?-synuclein mediated diseases or diseases in which ?-synuclein fibrils are a symptom or cause of the disease. In one embodiment, the compounds for use in the compositions and methods are heteroaryl acylguanidines, heteroarylhydrazones, dihydropyridones, heteroaryl and aryl styryl ketones, and heteroarylpyrazoles.
Type:
Grant
Filed:
September 16, 2005
Date of Patent:
May 14, 2013
Assignees:
Whitehead Institute for Biomedical Research, FoldRx Pharmaceuticals, Inc.
Inventors:
Susan L. Lindquist, Tiago Outeiro, Richard Labaudinière
Abstract: Compounds, compositions and methods are provided for stabilizing transthyretin and for treating, preventing, or ameliorating one or more symptoms of transthyretin mediated diseases. In one embodiment, the compounds are benzoxazoles and related compounds.
Abstract: Compounds, compositions and methods are provided for stabilizing transthyretin and for treating, preventing, or ameliorating one or more symptoms of transthyretin mediated diseases. In one embodiment, the compounds are benzoxazoles and related compounds.
Abstract: Compounds and compositions are provided for treatment or amelioration of one or more disorders characterized by defects in protein trafficking. A method of treating a disorder characterized by impaired protein trafficking includes administering to a subject or contacting a cell with a compound of Formula I: [formula here] or pharmaceutically acceptable salts or derivatives thereof.
Type:
Application
Filed:
November 7, 2008
Publication date:
December 30, 2010
Applicant:
FOLDRX PHARMACEUTICALS, INC.
Inventors:
Christine Ellen Bulawa, Michael DeVit, Daniel Elbaum
Abstract: Disclosed are compositions and methods for modulating protein trafficking and treating or preventing disorders characterized by impaired protein trafficking. Also disclosed are methods for producing a protein and identifying compounds that rescue protein trafficking defects.
Abstract: Compounds and compositions are provided for treatment or amelioration of one or more symptoms of ?-synuclein toxicity, ?-synuclein mediated diseases or diseases in which ?-synuclein fibrils are a symptom or cause of the disease.
Type:
Application
Filed:
March 29, 2007
Publication date:
October 28, 2010
Applicants:
FOLDRx PHARMACEUTICALS, INC, WHITEHEAD INSTITUTE FOR BIOMEDICAL RESEARCH
Inventors:
Susan L. Lindquist, Tiago Outeiro, Richard Labaudinière, James Fleming, Christine Ellen Bulawa, Charlotte Weigel, Feng Liang, Sandeep Gupta, Amy Ripka
Abstract: Disclosed are mammalian neuronal cells expressing alpha synuclein as well as methods of screening to identify compounds that decrease toxicity induced by alpha synuclein expression. Compounds identified by such screens can be used to treat or prevent synucleinopathies such as Parkinson's disease, familial Parkinson's disease, Lewy body disease, the Lewy body variant of Alzheimer's disease, dementia with Lewy bodies, multiple system atrophy, or the Parkinsonism-dementia complex of Guam.
Abstract: Disclosed are compositions and methods for modulating protein trafficking and treating or preventing disorders characterized by impaired protein trafficking. Also disclosed are methods for identification of compounds that rescue protein trafficking defects and methods of enhancing protein production.
Abstract: Compounds and compositions are provided for treatment or amelioration of one or more symptoms of ?-synuclein toxicity, ?-synuclein mediated diseases or diseases in which ?-synuclein fibrils are a symptom or cause of the disease. In one embodiment, the compounds for use in the compositions and methods are heteroaryl acylguanidines, heteroarylhydrazones, dihy-dropyridones, heteroaryl and aryl styryl ketones, and heteroarylpyrazoles.
Type:
Application
Filed:
September 16, 2005
Publication date:
October 23, 2008
Applicants:
WHITEHEAD INSTITUTE FOR BIOMEDICAL RESEARCH, FOLDRX PHARMACEUTICALS, INC.
Inventors:
Susan L. Lindquist, Tiago Outeiro, Richard Labaudiniere
Abstract: This invention relates to assays for detecting native-state proteins and to screening methods to identify compounds that modulate the stability of the native-state of proteins. Compounds identified by such screens can be used to treat diseases associated with protein misfolding, such as Alzheimer's Disease, familial amyloidotic polyneuropathies, and lysosomal storage diseases.