Diseases Treated
Inborn Errors of Metabolism
Adrenoleukodystrophy
Bare-lymphocyte syndrome
Dyskeratosis congenita
Familial erythrophagocytic lymphohistiocytosis
Gaucher disease
Gunter disease
Hunter syndrome
Hurler syndrome (genetic)
Inherited neuronal ceroid lipofuscinosis
Krabbe disease
Langerhans’-cell histiocytosis
Lesch-Nyhan disease
Leukocyte adhesion deficiency
Osteopetrosis (genetic) |
