A new investigation has identified a seemingly successful treatment for<unk>  by targeting CTLA4. Abatacept, an approved drug for rheumatoid arthritis, mimics the function of CTLA4 and has found to reverse life-threatening symptoms. The study included nine patients that exhibited improved clinical status and halted inflammatory conditions with minimal infectious or autoimmune complications. The study also suggests that therapies like chloroquine or hydroxychloroquine, which inhibit lysosomal degradation, may prove to be effective, as well. Larger cohorts are required to further validate these therapeutic approaches as effective long-term treatments for this disorder.
LRBA deficiency
NGLY1 deficiency
Adenosine deaminase 2 deficiency
WNT4 deficiency
Janus kinase 3 deficiency
PMM2 deficiency
Adenosine deaminase deficiency
GATA2 deficiency