Get Instant Summarized Text (Gist) A new approach using personalized antisense oligonucleotides (ASOs) and patient-derived organoids offers promising treatment options for rare genetic diseases ...

An FDA advisory committee has given tentative backing to Sarepta’s gene therapy for Duchenne muscular dystrophy ... Sarepta already has three antisense drugs for DMD on the market – Exondys ...

an investigational gene therapy designed to treat Duchenne muscular dystrophy (Duchenne). SGT-003 is a next-generation gene therapy candidate utilizing AAV-SLB101, a proprietary capsid developed ...