PEG-ADA replacement therapy for adenosine deaminase deficiency: an update after 8.5 years
MS Hershfield - Clinical immunology and immunopathology, 1995 - Elsevier
Polyethylene glycol-modified adenosine deaminase (PEG-ADA) has now been used for 8.5
years as enzyme replacement therapy for immunodeficiency due to ADA deficiency. PEG-
ADA restores a metabolic environment necessary for recovery of immune function. In most
cases, the level of function achieved has been sufficient to protect against opportunistic and
life-threatening infections. To date, mortality and morbidity with PEG-ADA have been less
than for haploidentical bone marrow transplantation. As a true “orphan drug” used to treat a …
years as enzyme replacement therapy for immunodeficiency due to ADA deficiency. PEG-
ADA restores a metabolic environment necessary for recovery of immune function. In most
cases, the level of function achieved has been sufficient to protect against opportunistic and
life-threatening infections. To date, mortality and morbidity with PEG-ADA have been less
than for haploidentical bone marrow transplantation. As a true “orphan drug” used to treat a …