Table 1 Effectiveness and safety outcomes

Laboratory assessments:

• Serum oligosaccharides (µmol/L)

• Serum IgG, IgA, and IgM

Functional assessments:

• 3MSCT (steps/min)

• 6MWT (m)^a

• 2MWT (m)^a

• FVC (L and % of predicted)^a

Health Assessment Questionnaires:

• EQ-5D-5L

• Zarit Burden Interview

• QoL questionnaire

• Behavior checklists for child and adult

Other:

• Psychotic events (rate)

• AEs: serious and nonserious

• ADRs: including serious and nonserious

• AEs leading to treatment discontinuation and death

• Any identified risks including anti-VA-IgG antibody, infusion-related reactions, and hypersensitivity

• Any potential risk of acute renal failure, loss of consciousness, and medication errors

• Vital signs: SBP, DBP, and pulse rate

• Electrocardiogram

• Laboratory tests (hematology and chemistry)

• Physical examination