Progressive familial intrahepatic cholestasis (PFIC)

Spinal muscular atrophy

Sjögren’s disease

PBC

Thyroid eye disease

Immunoglobulin G4-related disease (IgG4-RD)

Fabry disease

Hereditary amyloidosis (ATTRv)

Myelofibrosis

Glomerular diseases

acquired Thrombotic Thrombocytopenic Purpura (aTTP)

ANCA-associated vasculitis (AAV)

Activated PI3K delta syndrome (APDS)

Frontotemporal dementia

Gaucher

CDG

Acromegaly

Framework Implementation ACTION

Early Access