Zorevunersen appears safe and may alleviate symptoms in children with Dravet syndrome
1. In this phase 1/2a trial involving patients with Dravet syndrome who received intrathecal zorevunersen, treatment-related adverse events occurred in ...
Tag: phase 1 trial
Ex vivo gene therapy for cystinosis had acceptable safety and improved disease biomarkers
1. In this phase 1–2 open-label study, autologous lentiviral hematopoietic stem-cell gene therapy for cystinosis was associated with sustained engraftment ...
Safety and efficacy of novel antibody-oligonucleotide therapy for myotonic dystrophy type 1
1. In this phase 1-2 randomized controlled trial involving adults with myotonic dystrophy type 1, del-desiran was well-tolerated in most ...
Safety of investigational vaccines against human immunodeficiency virus
1. In this phase 1 randomized controlled trial, urticaria was reported in several participants who received a messenger RNA vaccine ...
