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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

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