Table 4 Summary of adverse events and serious adverse events.

From: Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons

 

Adverse eventsa

Serious adverse eventsb

Fatality

Related to gene therapy

  Skin irritation on cheeks due to dyskinesia

1

–

–

Related to study procedures

  Vomiting following administration of potassium iodide prior to DaTscan

1

–

–

  Post-operative anemia requiring transfusion

2

–

–

Related to AADC deficiency

  Sudden death of unknown cause

–

–

1

  Hypoglycemia following sedation for study procedure

2

1

–

Non-specific

  Viral respiratory tract infection

–

5

–

  Hematemesis associated with gastro-esophageal reflux

–

1

–

  Pneumonia

–

2

–

  Urinary tract infection

–

1

–

  Skin rash

2

–

–

  Eye infection/irritation

2

–

–

  Gastroenteritis

1

–

–

  Vomiting due to missed doses of anti-reflux medication

1

–

–

Total

12

10

1

  1. AADC aromatic l-amino acid decarboxylase deficiency.
  2. AADC is a rare neurodevelopmental disorder. Here the authors describe a clinical trial of MR-guided delivery of AAV2-AADC for the treatment of AADC.
  3. aAdverse events: classified as mild or moderate.
  4. bSerious adverse events: classified as severe or medically significant, requiring or prolonging hospitalization.
  5. Aromatic L-amino acid decarboxylase deficiency (AADC) is a rare neurodevelopmental disorder. Here the authors describe a clinical trial of MR-guided delivery of AAV2-AADC for the treatment of AADC.
Back to article page