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aHUS – atypical hemolytic uremic syndrome
Amyloidosis
gMG – generalized myasthenia gravis
HPP – hypophosphatasia
LAL-D – lysosomal acid lipase deficiency
NF1 — neurofibromatosis type 1 (NF1) plexiform neurofibromas (PN)
NMOSD — neuromyelitis optica spectrum disorder
PNH — paroxysmal nocturnal hemoglobinuria
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