- The Foundation for the National Institutes of Health announces the selection of eight rare diseases for the Bespoke Gene Therapy Consortium’s clinical trial portfolioThe National Institutes of Health Foundation
- NIH Foundation Adds 8 Rare Diseases to Clinical Trials PortfolioFierceBiotech
- Nemours Children’s Health Selected to Conduct First Gene Therapy Clinical Trial for Morquio A SyndromeNews-Medical.Net
- ASGCT News: Eight rare diseases selected for new pipeline of gene therapy clinical trialsGenetic Engineering and Biotechnology News