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NON-IMMUNOSUPPRESSOR CYCLOSPORIN FOR TREATMENT OF CONGENITAL MYOPATHY OF ULRICH
NON-IMMUNOSUPPRESSOR CYCLOSPORIN FOR TREATMENT OF CONGENITAL MYOPATHY OF ULRICH
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机译:非免疫抑制环氯嘌呤治疗乌尔里希先天性肌病
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摘要
1. The use of the non-immunosuppressive derivative of cyclosporin A of formula I to reduce mitochondrial dysfunction and the degree of apoptosis in the cells of patients suffering from congenital Ulrich myopathy or Betlem myopathy. ! 2. The use according to claim 1, where the non-immunosuppressive derivative of cyclosporin A is a derivative of cyclosporin of the formula II. ! 3. The use according to claim 1, where the non-immunosuppressive derivative of cyclosporin A is a non-immunosuppressive cyclosporin [D-MeAla] 3- [EtVal] 4-CsA of formula III. ! 4. The use of the non-immunosuppressive derivative of cyclosporin A of formula I for the manufacture of a medicament for the treatment of congenital Ulrich myopathy or Betlem myopathy. ! 5. The use according to claim 4, where the non-immunosuppressive derivative of cyclosporin A is a derivative of cyclosporin of the formula II. ! 6. The use according to claim 4, where the non-immunosuppressive derivative of cyclosporin A is a non-immunosuppressive cyclosporin [D-MeAla] 3- [EtVal] 4-CsA of formula III. ! 7. A method for treating Ulrich’s congenital myopathy or Betlem’s myopathy in patients in which an effective amount of a non-immunosuppressive cyclosporin A derivative of formula I is administered to the patient! 8. The method according to claim 7, where the non-immunosuppressive derivative of cyclosporin A is a derivative of cyclosporin of the formula II. ! 9. The use according to claim 7, where the non-immunosuppressive derivative of cyclosporin A is a non-immunosuppressive cyclosporin [D-MeAla] 3- [EtVal] 4-CsA of formula III. ! 10. A pharmaceutical composition for treating Ulrich’s congenital myopathy or Betlem’s myopathy, comprising an effective amount of a non-immunosuppressive cyclosporin A derivative of formula I, a pharmaceutically acceptable carrier and, optionally, an excipient and diluent. !eleven
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