Patients affected by ADA-SCID have compromised immune systems that leave them unprotected from infection-producing bacteria, viruses, and fungi.
Undiagnosed babies with ADA-SCID usually die before they reach age two due to infections.
SCID newborn screening in most states has allowed detection of ADA-SCID in newborns and has led to early initiation of ADA enzyme therapy and improved outcomes.
Ada-SCID affects both boys and girls and is more difficult to treat than X-SCID.
A bone marrow transplant from a good donor can cure ada-SCID 90pc of the time.
So far, more than 40 ADA-SCID
patients have been treated with autologous ex-vivo lentiviral gene therapy at GOSH and UCLA.
To date, over 40 ADA-SCID
patients have been treated in clinical trials with autologous ex-vivo lentiviral gene therapy at UCLA, Los Angeles and at the Great Ormond Street Hospital (GOSH) in London, UK.
is a very rare and life-threatening disease that affects approximately 350 children worldwide.
is a very rare and life-threatening disease caused by the alteration of a single gene and affects approximately 350 children worldwide.