Diagnostic challenges and new therapies for rare lysosomal storage diseases.
Hesselgrave
Barbara
B
64mgb@shentel.net
D019090:Case Management; D015984:Causality; D019468:Disease Management; D018450:Disease Progression; D000795:Fabry Disease / Q000175:diagnosis* / Q000453:epidemiology / Q000209:etiology / Q000628:therapy*; D006801:Humans; D007527:Isoenzymes / Q000627:therapeutic use; D008059:Mucopolysaccharidosis I / Q000175:diagnosis* / Q000453:epidemiology / Q000209:etiology / Q000628:therapy*; D009965:Orphan Drug Production; D035583:Rare Diseases / Q000175:diagnosis* / Q000453:epidemiology / Q000209:etiology / Q000628:therapy*; D013997:Time Factors; D014481:United States / Q000453:epidemiology; D000519:alpha-Galactosidase / Q000627:therapeutic use
D007527:Isoenzymes; D000519:alpha-Galactosidase; C459420:agalsidase beta