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Transthyretin-mediated amyloidosis (ATTR amyloidosis)

Central Nervous System/Neurodegenerative Diseases
Examples: Alzheimer's, Huntington's, Parkinson's, Amyotrophic Lateral Sclerosis (ALS)

Primary Hyperoxaluria Type 1

Porphyria

Complement-Mediated Diseases
Examples: Atypical Hemolytic Uremic Syndrome (aHUS), Paroxysmal Nocturnal Hemoglobinuria (PNH),
IgA Nephropathy/Berger's

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