[EN] COMPOUNDS AND METHODS FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY<br/>[FR] COMPOSÉS ET PROCÉDÉS POUR LE TRAITEMENT DE LA DYSTROPHIE MUSCULAIRE DE DUCHENNE
申请人:DTX PHARMA INC
公开号:WO2021108640A1
公开(公告)日:2021-06-03
Disclosed herein are compounds including a single-stranded oligonucleotide (A) having a nucleobase sequence complementary to a portion of the dystrophin pre-mRNA, their preparation, and uses thereof for the treatment of Duchenne muscular dystrophy.