Means and methods for counteracting muscle disorders
First Claim
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1. A composition comprising:
- a first compound for providing an individual with a functionaldystrophin protein; and
a second compound;
wherein said second compound comprises at least one of;
a compound for reducing inflammation, anda compound for improving muscle fiber function, integrity and/or survival.
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Abstract
The invention provides means and methods for alleviating one or more symptom(s) of Duchenne Muscular Dystrophy and/or Becker Muscular Dystrophy. Therapies using compounds for providing patients with functional muscle proteins are combined with at least one adjunct compound for reducing inflammation, preferably for reducing muscle tissue inflammation, and/or at least one adjunct compound for improving muscle fiber function, integrity and/or survival.
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Citations
41 Claims
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1. A composition comprising:
- a first compound for providing an individual with a functional
dystrophin protein; and
a second compound;wherein said second compound comprises at least one of; a compound for reducing inflammation, and a compound for improving muscle fiber function, integrity and/or survival. - View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, 20)
- a first compound for providing an individual with a functional
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21. A method for alleviating one or more symptom(s) of Duchenne Muscular Dystrophy or Becker Muscular Dystrophy in an individual, the method comprising:
administering to said individual;
a first compound that provides a functional dystrophin protein, and a second compound,wherein said second compound comprises at least one of; a compound that reduces inflammation, and/or a compound that improves muscle fiber function, integrity and/or survival. - View Dependent Claims (22, 23, 24, 25, 26, 27, 28, 29, 30, 31, 32, 33, 34, 35)
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36. A method for increasing the production of a functional dystrophin protein in a cell, said cell comprising pre-mRNA of a dystrophin gene encoding an aberrant dystrophin protein comprising:
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providing said cell with a first compound for inhibiting inclusion of an exon into mRNA produced from splicing of said dystrophin pre-mRNA, and providing said cell with a second compound; wherein said second compound comprises at least one of; a compound that reduces inflammation, and/or a compound for improving muscle fiber function, integrity and/or survival, said method further comprising allowing translation of mRNA produced from splicing of said pre-mRNA. - View Dependent Claims (37)
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38. A pharmaceutical preparation comprising:
- a first compound for providing an individual with a functional dystrophin protein, and a second compound, wherein said second compound comprises at least one of;
a compound for reducing inflammation, and a compound for improving muscle fiber function, integrity and/or survival, and a pharmaceutically acceptable carrier, adjuvant, diluent and/or excipient.
- a first compound for providing an individual with a functional dystrophin protein, and a second compound, wherein said second compound comprises at least one of;
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39. A kit comprising a first compound for providing an individual with a functional dystrophin protein, and a second compound, wherein said second compound comprises at least one of:
- a compound for reducing inflammation, and a compound for improving muscle fiber function, integrity and/or survival.
- View Dependent Claims (40, 41)
Specification