Genetic disorders affecting multiple organs constitute 4.4% of the trials, addressing a variety of inherited conditions, including hereditary angioedema, transthyretin-related familial amyloid polyneuropathy, transthyretin-related familial amyloid cardiomyopathy, wild-type transthyretin cardiac amyloidosis, Kabuki syndrome 1, Rubinstein-Taybi syndrome, and pyruvate kinase deficiency. Here, TTR is linked to Kabuki syndrome 1.