Ann: Half Year Financial Results Presentation, page-64

More an answer to the doubters than your post above @reginaldp. Adult EAP survival figures would be considered real world evidence, which the FDA increasingly leans on. Remember, too that this adult trial is going to be conducted by the Bone Marrow Transport - Clinical Trial Network (funded by the National Institute of Health) which handles 80% of the cases, with a trove of clinical data from other developers of drugs to treat the condition, as well as biomarker data which can be used to tailor the trial to people whose inflammation makes them best candidates.