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Exon skipping therapy of erythropoietic protoporphyria

The present invention relates to methods and pharmaceutical compositions for the treatment of Erythropoietic Protoporphyria. In particular, the […]

Hydrophobically modified antisense oligonucleotides comprising a cetal

The present invention concerns an oligonucleotide modified by substitution at the 3’ or the 5’ end by at least triple alkyl chain or at least one […]

New antisense oligonucleotide targeting all cystic fibrosis patients

The present invention relates to methods and pharmaceutical compositions for the treatment of cystic fibrosis (CF). In particular, the present […]

Lipid antisens oligonucleotide targeting fxyd2 as a therapeutic agent

The present invention relates to an inhibitor of FXYD2 gene expression for use in a method for treating neuropathic pain in a patient in need thereof. […]