Sialic acid analogs
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Accused Products
Abstract
The present invention provides sialic acid analogs and their compositions useful for the treatment of sialic acid deficiencies.
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Citations
21 Claims
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1. A compound having structural Formula (Ia):
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2. The compound of claim 1, wherein R3a is C1 to C20 alkyl.
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3. The compound of claim 2, wherein structural Formula (Ia) is represented by structural Formula (IIa):
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4. The compound of claim 1,
wherein R1, R2, and R4 are hydrogen; - and
R6 is structural formula (f);
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5. The compound of claim 4, wherein R6 is structural formula (f);
- and R3a is C1 to C20 alkyl.
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6. The compound of claim 4, wherein R6 is structural formula (f);
- and R3a is C1 to C12 alkyl.
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7. The compound of claim 1, which is selected from the group consisting of
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8. A pharmaceutical composition comprising a compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof, and a pharmaceutically acceptable carrier.
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9. A sustained release pharmaceutical composition comprising a compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof, wherein the release of the compound is over a period of about four hours or more.
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10. A sustained release pharmaceutical composition comprising a compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof, wherein the pharmacological effect from the compound lasts about four hours or more upon administration of the composition.
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11. A sustained release pharmaceutical composition comprising a compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof;
wherein the composition, upon administration, provides a therapeutically effective amount of the compound for about 4 hours or more.
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12. The sustained release pharmaceutical composition of claim 7, comprising a matrix which comprises
a compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof; - and
one or more release rate controlling polymers.
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13. The sustained release pharmaceutical composition of claim 12, wherein the matrix is in form of a core or a layer over a core.
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14. The sustained release pharmaceutical composition of claim 7, comprising a release rate controlling membrane disposed over:
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a pull layer comprising a compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof, and an osmotic push layer; wherein the release rate controlling membrane has an orifice immediately adjacent to the pull layer.
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15. The sustained release pharmaceutical composition of claim 12, wherein the pull layer further comprises a release rate controlling polymer.
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16. The sustained release pharmaceutical composition of claim 7, comprising one or more particles, and each of the particles comprises
an active core comprising a compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof; - and
a release rate controlling polymer disposed over the core.
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17. The sustained release pharmaceutical composition of claim 7, comprising one or more particles, and each of the particles comprises
an inert core, an active layer comprising a compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof disposed over the inert core, and a release rate controlling polymer disposed over the active layer.
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18. A method for treating a sialic acid deficiency in a patient in need thereof comprising administering an effective amount of a compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof.
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19. A method for treating a sialic acid deficiency in a patient in need thereof comprising administering a compound of claim 1, or a pharmaceutically acceptable salt or solvate thereof;
- wherein upon administration, the compound, or a pharmaceutically acceptable salt or solvate thereof, continuously provides a therapeutically effective amount of sialic acid for about 4 hours to about 24 hours.
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20. The method of claim 18, wherein the sialic acid deficiency is a myopathy associated with sialic acid deficiency.
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21. The method of claim 20, wherein the myopathy associated with sialic acid deficiency is Hereditary Inclusion Body Myopathy (HIBM), Nonaka myopathy, and/or Distal Myopathy with Rimmed Vacuoles (DMRV).
Specification