发明公开
EP1054058A1 Pharmaceutical composition for treatment of Duchenne muscular dystrophy 审中-公开
Pharmazutische Zusammensetzung zur Behandlung von Duchenne-Muskeldystrophie

Pharmaceutical composition for treatment of Duchenne muscular dystrophy
摘要:
A therapeutic pharmaceutical composition for patients of Duchenne muscular dystrophy with entire loss of exon 20 in dystrophin mature mRNA is provided. The composition comprise as an active principle an antisense oligonucleotide consisting of a 20 to 50-nucleotide sequence against exon 19 of the dystrophin pre-mRNA.
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