Personalized therapy for rare genetic diseases: Patient-derived organoids offer new hope
Children's Mercy Kansas City has achieved a significant advancement toward the treatment of rare genetic diseases through the ...
pharmaphorum1mon
FDA advisors narrowly back Sarepta’s Duchenne gene therapy
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 ...
The American Journal of Managed Care6h
EMBARK 2: Delandistrogene Moxeparvovec Shows Sustained Benefits in DMD
The gene therapy delandistrogene moxeparvovec-rokl showed clinically meaningful benefits and disease stabilization at 2 years ...
MedCity News1y
Genetic Medicines Biotech Dyne Pulls In $345M for Clinical Tests of Muscle Drugs
Dyne CEO Joshua Brumm said that this year the Phase 1/2 studies in myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy ... therapies based on antisense oligonucleotides, nucleic ...
Research explores genetic treatment for rare arrhythmias
Although ASOs targeting liver cells are in clinical use and highly effective, so far the only oligonucleotide therapies ...
Nasdaq12d
Solid Biosciences Initiates Clinical Trial For Duchenne Therapy SGT-003
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 ...