DAVID H PERLMUTTER
Medical Practice in Pittsburgh, PA

6403646, Jun 11, 2002

Jun 30, 2000

09/608324

David H. Perlmutter - Pittsburgh PA 15213
Jon A. J. Burrows - St. Louis MO 63110
Lauren K. Willis - Norfolk VA 23510-1001
Jeffery H. Teckman - St. Louis MO 63110

A61K 31192

514570, 514569, 514568

A method for the treatment of alpha-1-antitrypsin deficiency caused by the protease inhibitor type Z mutation by administration of phenylbutyric acid derivatives. Also disclosed are methods for the prevention and treatment of liver injury and emphysema associated with alpha-1-antitrypsin deficiency by administration of phenylbutyric acid derivatives.

2013002, Jan 24, 2013

May 3, 2012

13/463638

Stephen C. Pak - Pittsburgh PA, US
David Hirsch Perlmutter - Pittsburgh PA, US
Gary A. Silverman - Pittsburgh PA, US

University of Pittsburgh - of the Commonwealth System of Higher Education - Pittsburgh PA

A61K 31/454, A61P 25/16, A61K 31/5415, A61K 31/138, A01K 67/033, A61K 31/34, A61K 31/704, A61K 31/545, C07H 21/00, A61K 49/00, A61P 25/28, A61K 31/337

800 3, 514322, 5142258, 514646, 514449, 514468, 514 27, 514209, 536 235, 800 9, 800 12

The present invention relates to methods of treatment of clinical disorders associated with protein aggregation comprising administering, to a subject, an effective amount of an anti-protein aggregate (“APA”) compound selected from the group consisting of pimozide, fluphenazine (e.g., fluphenazine hydrochloride), tamoxifen (e.g., tamoxifen citrate), taxol, cantharidin, cantharidic acid, salts thereof and their structurally related compounds. It is based, at least in part, on the discovery that each of the aforelisted compounds were able to promote degradation of aggregated ATZ protein in a model system. According to the invention, treatment with one or more of these APA compounds may be used to ameliorate the symptoms and signs of AT deficiency as well as other disorders marked by protein aggregation, including, but not limited to, Alzheimer's Disease, Parkinson's Disease, and Huntington's Disease.

2012012, May 24, 2012

Jan 31, 2012

13/362606

David Hirsch Perlmutter - Pittsburgh PA, US
George Konstantine Michalopoulos - Pittsburgh PA, US
Tunde Hidvegi - Gibsonia PA, US

A61K 31/55, A61P 25/16, A61P 25/28

514217

The present invention relates to methods of treatment of clinical disorders associated with protein polymerization comprising administering, to a subject, an effective amount of carbamazepine, oxcarbazepine or another carbamazepine-like compound. It is based, at least in part, on the discovery that, in cells having a genetic defect in α1-antitrypsin, carbamazepine was able to decrease levels of the mutant protein. Furthermore, carbamazepine reduced the hepatic load of mutant α1-antitrypsin and the toxic effect of that mutant protein accumulation, hepatic fibrosis, in vivo using a mouse model of the disease. As patients having this defect in α1-antitrypsin exhibit toxic accumulations of the protein, treatment according to the invention may be used to ameliorate symptoms and signs of disease.

2011015, Jun 23, 2011

Sep 14, 2010

12/881976

Stephen C. Pak - Pittsburgh PA, US
David H. Perlmutter - Pittsburgh PA, US
Gary A. Silverman - Pittsburgh PA, US

G01N 33/00, C07H 21/04, C12N 15/63, A01K 67/00

800 3, 536 231, 4353201, 800 13, 800 9, 800 12

The present invention relates to methods and compositions for high content drug screening in which may be used to identify compounds that treat disorders associated with protein aggregation.