Disease Subtype(s) HCT Disease Inserts Cellular Therapy Disease Inserts
  • Osteopetrosis(malignant infantile osteopetrosis) (521)
Forms 2036 & 2136 No disease inserts required
Leukodystrophies
  • Metachromatic leukodystrophy (MLD) (542)
  • Adrenoleukodystrophy (ALD) (543)
  • Krabbe disease (globoid leukodystrophy) (544)
Forms 2037 & 2137
  • Lesch-Nyhan (HGPRT deficiency) (522)
  • Neuronal ceroid lipofuscinosis (Batten disease) (523)
Mucopolysaccharidosis
  • Hurler syndrome (IH) (531)
  • Scheie syndrome (IS) (532)
  • Hunter syndrome (II) (533)
  • Sanfilippo (III) (534)
  • Morquio (IV) (535)
  • Maroteaux-Lamy (VI) (536)
  • β-glucuronidase deficiency (VII) (537)
  • Mucopolysaccharidosis (V) (538)
  • Mucopolysaccharidosis, not otherwise specified (530)
Mucolipidoses
  • Gaucher disease (541)
  • Niemann-Pick disease (545)
  • I-cell disease (546)
  • Wolman disease (547)
  • Glucose storage disease (548)
  • Mucolipidoses, not otherwise specified (540)
Polysaccharide Hydrolase Abnormalities
  • Aspartyl glucosaminidase (561)
  • Fucosidosis (562)
  • Mannosidosis (563)
  • Polysaccharide hydrolase abnormality, not otherwise specified (560)
  • Other inherited metabolic disorder (529)
  • Inherited metabolic disorder, not otherwise specified (520)
Forms 2038 & 2138
Last modified: May 29, 2024