COMPOSITIONS AND METHODS FOR TREATING DUCHENNE MUSCULAR DYSTROPHY AND RELATED DISORDERS
申请人:Sarepta Therapeutics, Inc.
公开号:EP3858993A1
公开(公告)日:2021-08-04
The present disclosure relates to compositions and methods for the treatment of Duchenne muscular dystrophy and related disorders. Modified antisense oligomers are disclosed for the treatment of Duchenne muscular dystrophy and related disorders.
本公开涉及治疗杜氏肌营养不良症及相关疾病的组合物和方法。本发明公开了用于治疗杜氏肌营养不良症及相关疾病的修饰反义寡聚物。