Compositions and methods for the treatment of cystic fibrosis
OwnerDouglas V. Faller, Susan P. Perrine +1inventors
InventorsDouglas V. Faller, Susan P. Perrine, George Stamatoyannopoulos
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OwnerDouglas V. Faller, Susan P. Perrine +1inventors
InventorsDouglas V. Faller, Susan P. Perrine, George Stamatoyannopoulos
Estimating the term…
The invention is directed to novel pharmaceutical compositions comprising chemicals agents that are useful in the treatment and prevention of cystic fibrosis and the prevention of signs and symptoms of this disease. These pharmaceutical compositions are surprisingly successful in the treatment disorders related to cystic fibrosis including disorders of blood production. Many of these compositions of the invention are even more effective when administered to a patient in pulses. Pulse therapy is not a form of discontinuous administration of the same amount of a composition over time, but comprises administration of the same dose of the composition at a reduced frequency or administration of reduced doses.
A method for the treatment or prevention of cystic fibrosis comprising the administration of a composition comprising a physiologically-effective amount of one or more agents selected from the group consisting of butyric acid ethyl ester, 2,2-dimethyl butyric acid, 2,2-diethyl butyric acid, 3,3-dimethyl butyric acid, 3,3-diethyl butyric acid, 2,3-dimethyl succinic acid, methoxy acetic acid, phenoxyacetic acid, 2- and 3-thiophenoxy propionic acid, 2- and 3-phenoxy propionic acid, 2- and 3-phenyl propionic acid, 4-chlorophenoxy-2-propionic acid, methoxy acetic acid, or 2-thiophenoxy acetic acid, or a chemical compound of the structure phenyl-R9-R10 wherein R9 is CHx, CO, NHx, OHx, SHx, or a branched or linear aryl chain;
A method for the therapy of cystic fibrosis comprising administering to a patient a quantity of an agent, or pharmaceutically acceptable derivatives thereof, effective for said therapy, said agent selected from the group consisting of butyric acid ethyl ester, 2,2-dimethyl butyric acid, 2,2-diethyl butyric acid, 3,3-dimethyl butyric acid, 3,3-diethyl butyric acid, 2,3-dimethyl succinic acid, methoxy acetic acid, phenoxyacetic acid, 2- and 3-thiophenoxy propionic acid, 2- and 3-phenoxy propionic acid, 2- and 3-phenyl propionic acid, 4-chlorophenoxy-2-propionic acid, methoxy acetic acid, 2-thiophenoxy acetic acid, or a chemical compound of the structure phenyl-R9—R10 wherein R9 is CHx, CO, NHx, OHx, SHx, or a branched or linear aryl chain;
A method for enhancing expression of CFTR comprising the administration of a physiologically effective amount of one or more agents or pharmaceutically acceptable derivatives thereof, said agents selected from the group consisting of butyric acid ethyl ester, 2,2-dimethyl butyric acid, 2,2-diethyl butyric acid, 3,3-dimethyl butyric acid, 3,3-diethyl butyric acid, 2,3-dimethyl succinic acid, methoxy acetic acid, phenoxyacetic acid, 2- and 3-thiophenoxy propionic acid, 2- and 3-phenoxy propionic acid, 2- and 3-phenyl propionic acid, 4-chlorophenoxy-2-propionic acid, methoxy acetic acid, 2-thiophenoxy acetic acid, and chemical compounds of the structure phenyl-R9—R10 wherein R9, is CHx, CO, NHx, OHx, SHx, or a branched or linear aryl chain;
Dependencies are inferred from the claim text.