A gene therapy treatment is being used to treat X-linked severe combined immunodeficiency (X-SCID). Mustang Bio has licensed the treatment from St. Jude Children’s Research Hospital. X-SCID is also known as “bubble boy” syndrome in which there are mutations in genes that develop immune cells. The treatment involves giving X-SCID patients busulfan (a low dose cancer drug) before reinfusing them with their own gene modified stem cells.
Read more at the source article fiercebiotech.com
© Copyright Patient Worthy
Sign Up With a Patient Worthy Account and Share Your Rare Story
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
- OR -
Make a difference, share your experiences and get paid. Opt-in and join Patient Worthy's panel for paid opportunities such as surveys, market research, patient advisory panels and more.
