Grade 1–2 adverse events occurred in 12 cases with a median of 1 adverse event (range 0–5) per individual and included mild transient stomatitis (2 cases), worsening of infantile acne (1 case), increase of serum cholesterol and triglycerides (4 cases), changes in serum phosphate levels (2 cases), increase of cholinesterase (2 cases), increase of serum lactate dehydrogenase (1 case), transient neutropenia (2 cases), transient anemia (1 case), transient lymphopenia (1 case) and recurrent infections (7 cases) (Fig. 2). The gene discussed is BCHE; the disease is anemia.