PATENT: METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE

Bibliographic data: US2016024526 (A1) ― 2016-01-28
Inventor(s): PUCCIO HELENE MONIQUE [FR]; AUBORG PATRICK [FR]; CRYSTAL RONALD G [US]; BOUGNERES PIERRE [FR]
Applicant(s): APHP ASSISTANCE PUBLIQUE HÔPITAUX DE PARIS [FR]; UNIV CORNELL [US]; INST NAT SANTE RECH MED [FR]; CENTRE NAT RECH SCIENT [FR]; UNIV STRASBOURG [FR]; UNIVERSITÉ PARIS SUD XI [FR]

The invention relates to a method for preventing or treating a cardiomyopathy associated with Friedreich ataxia in a subject in need thereof, comprising administering to said subject a therapeutically effective amount of a vector which comprises a frataxin (FXN) encoding nucleic acid.


METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE