Table 4 Overall distribution of onasemnogene abeparvovec-related AEs

Investigations

1889

aspartate aminotransferase increased (284); alanine aminotransferase increased (260); hepatic enzyme increased (237); platelet count decreased (140); liver function test increased (116); troponin i increased (113); transaminases increased (87); troponin increased (83); blood lactate dehydrogenase increased (71); oxygen saturation decreased (60); body temperature increased (39);

troponin t increased (33); white blood cell count decreased (33); blood creatine phosphokinase increased (30); gamma-glutamyltransferase increased (30); platelet count increased (30); white blood cell count increased (29);

heart rate increased (28); blood bilirubin increased (22);

monocyte count increased (22); C-reactive protein increased (21); blood creatinine decreased (19); weight decreased (45); weight increased (24); haemoglobin decreased (17);

blood creatinine increased (16).

Gastrointestinal disorders

737

vomiting (434); dysphagia (52); constipation (39); salivary hypersecretion (22); retching (17); abdominal distension (19); nausea (67); diarrhoea (55); gastrooesophageal reflux disease (17).

General disorders and administration site conditions

705

pyrexia (461); illness (24); crying (17); fatigue (46); drug ineffective (36); death (35); asthenia (27); malaise (24);

feeling abnormal (19); general physical health deterioration (15).

Respiratory, thoracic and mediastinal disorders

521

cough (86); respiratory failure (44); respiratory distress (37); rhinorrhoea (36); respiratory disorder (34); atelectasis (33); increased bronchial secretion (26); aspiration (20);

hypoxia (20); productive cough (19); tachypnoea (19); acute respiratory failure (18); pneumothorax (18); choking (15); dyspnoea (96).

Infections and infestations

433

pneumonia (112); rhinovirus infection (46); covid-19 (44);

nasopharyngitis (40); respiratory syncytial virus infection (40); pneumonia aspiration (29); respiratory tract infection (27); upper respiratory tract infection (23); viral infection (19); lower respiratory tract infection (18); bronchiolitis (15); influenza (20).

Blood and lymphatic system disorders

283

thrombocytopenia(170); thrombotic microangiopathy(35); neutropenia(26); leukopenia(27); anaemia(25).

Metabolism and nutrition disorders

146

decreased appetite(95); dehydration(35); hyperkalaemia(16).

Cardiac disorders

106

tachycardia(50); bradycardia(33); cardiac arrest(23);

Renal and urinary disorders

66

proteinuria(28); haematuria(21); renal impairment(17).

Psychiatric disorders

68

irritability(53); eating disorder(15).

Nervous system disorders

75

somnolence(25); lethargy(29); tremor(21).

Musculoskeletal and connective tissue disorders

58

scoliosis(30); muscular weakness(28).

Hepatobiliary disorders

64

hepatic function abnormal(33); hypertransaminasaemia(31).

Vascular disorders

70

pallor (16); cyanosis (15); hypertension (39).

Skin and subcutaneous tissue disorders

32

rash(32).