We know rare. We are rare.
Acute Hepatic Porphyria
Alport Syndrome
Amyotrophic Lateral Scerosis
Aatypical Hemolytic Uremic Syndrome (aHUS)
C3 Glomerulopathy hATTR Amyloidosis
Chronic Lymphocytic Leukemia
Chronic Myelogenous Leukemia
Common Variable Immunodeficiency
Congenital TTP
Cystic Fibrosis
Duchenne Muscular Dystrophy
Fabry Disease
Familial Chylomicronemia Syndrome
Gaucher Disease
Hemophilia A
Hemophilia B
Homozygous Familial Hypercholesterolemia
Hypoparathyroidism
Lipodystrophy
Mantle Cell lymphoma
MPS I, II (Mucopolysaccharidosis )
Myasthenia Gravis
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Polycythemia Vera
Pompe Disease
Primary Immunodeficiency
Pyruvate Kinase Deficiency
Sickle Cell Disease
Spinal Muscular Atrophy
Tuberous Sclerosis
Von Willebrand disease
WHIM Syndrome
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