Sentences with phrase «bubble boy syndrome»
The company is also seeking deeper inroads into rare diseases, with a number of assets in late - stage development, and last year it scored a major regulatory win when Europe approved its «bubble boy syndrome» gene therapy Strimvelis.
https://www.fiercebiotech.com/ Not exact matches
Scientists believe that is what happened during a 1999 French gene therapy trial on a group of 10 young children with X-SCID, an immune deficiency disorder known as boy - in - the - bubble syndrome.
Scientists believe that's what happened during a 1999 French gene therapy trial on a group of 10 infants with X-SCID, an immune deficiency disorder known as boy - in - the - bubble syndrome.