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US 8,575,222 B2Granted
Use of nitrooxyderivatives of drug for the treatment of muscular dystrophies
AssigneeNicox S.A.
InventorsEmilio Clementi, Giulio Cossu, Silvia Brunelli, Ennio Ongini
Life of this patent
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Abstract
The present invention relates to the use of nitric oxide releasing compounds for retarding or reversing muscular dystrophies such as Duchenne and Becker dystrophies.
Claims17
A method of treating muscular dystrophies comprising administering to a subject a nitric oxide releasing compound of formula (Ia)
- M-X—Y—ONO2 (Ia)
- or enantiomers or diastereoisomers thereof, wherein in the general formula (Ia), M, X and Y have the following meanings:
- M is a residue selected from the group consisting of:
- X is —O—, —S— or —NR1—, wherein R1 is H or linear or branched C1-C6 alkyl;
- Y is a bivalent radical having the following meanings:
- a)—straight or branched C1-C20 alkylene being optionally substituted with one or more of the substituents selected from the group consisting of:
- halogen atoms, hydroxy, —ONO2 and T, wherein T is —OC(O)(C1-C10 alkyl)-ONO2 or —O(C1-C10 alkyl) ONO2;
- C5-C7 cycloalkylene group optionally substituted with linear or branched C1-C10 alkyl group;
- wherein n is an integer from 0 to 20;
- n1 is an integer from 1 to 20 wherein when Y is selected from the bivalent radicals mentioned under b) and c) then the —ONO2 group of formula is bound to —(CH2)n1;
- wherein
- X1=—OCO— or —COO— and R2 is H or CH3;
- na is an integer from 1 to 20;
- n2 is an integer from 0 to 2;
- wherein:
- Y1 is —CH2—CH2—(CH2)n2—;
- or —CH═CH— (CH2)n2;
- X1, na, n2 and R2 are as defined above;
- wherein:
- na and R2 are as defined above, R3 is H or —COCH3;
- wherein when Y is selected from the bivalent radicals mentioned under d)-f) then the —ONO2 group of formula (I) is bound to —(CH2)na;
- wherein when X is —NR1—, wherein R1 is as above defined Y cannot be f);
- and
- wherein
- X2 is —O— or —S—;
- n3 is an integer from 1 to 6 and
- R2 is as defined above.
24 more elements
A method of treating muscular dystrophies consisting of administering to a subject a nitric oxide releasing compound of formula (Ia)
- M-X—Y—ONO2 (Ia)
- or enantiomers or diastereoisomers thereof, wherein in the general formula (Ia), M, X and Y have the following meanings:
- M is a residue selected from the group consisting of:
- X is —O—, —S— or —NR1—, wherein R1 is H or linear or branched
- C1-C6 alkyl;
- Y is a bivalent radical having the following meanings:
- a)—straight or branched C1-C20 alkylene being
- optionally substituted with one or more of the substituents selected from the group consisting of:
- halogen atoms, hydroxy, —ONO2 and T, wherein T is —OC(O)(C1-C10 alkyl)-ONO2 or —O(C1-C10 alkyl) ONO2;
- C5-C7 cycloalkylene group optionally substituted with linear or branched C1-C10 alkyl group;
- wherein n is an integer from 0 to 20;
- n1 is an integer from 1 to 20 wherein when Y is selected from the bivalent radicals mentioned under b) and c) then the —ONO2 group of formula is bound to —(CH2)n1—;
- wherein
- X1=—OCO— or —COO— and R2 is H or CH3;
- na is an integer from 1 to 20;
- n2 is an integer from 0 to 2;
- wherein:
- Y1 is —CH2—CH2—(CH2)n2—;
- or —CH═CH—(CH2)n2—;
- X1, na, n2 and R2 are as defined above;
- wherein:
- na and R2 are as defined above, R3 is H or —COCH3;
- wherein when Y is selected from the bivalent radicals mentioned under d)-f) then the —ONO2 group of formula (I) is bound to —(CH2)na;
- wherein when X is —NR1, wherein R1 is as above defined Y cannot be f);
- and
- wherein
- X2 is —O— or —S—;
- n3 is an integer from 1 to 6 and
- R2 is as defined above.
26 more elements
Dependencies are inferred from the claim text.