Q: Severe combined immunodeficiency (SCID) is characterized by severely impaired T-cell development and is fatal without treatment. What is the current treatment of infants identified as having SCID, and what is their likely clinical outcome if treated shortly after birth? A: • Overall, with improvements in...

Q: The CBER’s Regenerative Medicine Advanced Therapy Designation program has been very successful, with about 100 requests for designation in the two years of its existence. Can you please tell us about the program and how it was put together? A: The Regenerative Medicine Advanced Therapy...