Antibiotics for treating osteomyelitis in people with sickle cell disease (protocol stage)

Antioxidant micronutrients for lung disease in cystic fibrosis (protocol stage)

Bisphosphonate therapy for osteogenesis imperfecta (protocol stage)

Carnitine supplementation for treating people with inborn errors of metabolism (protocol stage)

Disease modifying anti-rheumatic drugs in people with cystic fibrosis-related arthritis (protocol stage)

Enzyme replacement therapy for Anderson-Fabry disease (protocol stage)

Hematopoietic stem cell transplantation for children with sickle cell disease (protocol stage)

Hematopoietic stem cell transplantation for Gaucher disease (protocol stage)

Inspiratory muscle training for cystic fibrosis (protocol stage)

Oscillating devices for airway clearance in people with cystic fibrosis (protocol stage)

Statins for familial hypercholesterolemia in children (protocol stage)

Thiol derivatives for pulmonary disease in cystic fibrosis (protocol stage)

Vaccines for preventing invasive salmonella infections in people with sickle cell disease (protocol stage)

Vitamin D supplementation for cystic fibrosis (protocol stage)