Advances in the treatment of lysosomal storage disease

This publication has 48 references indexed in Scilit:

• Comparative Efficacy of Dose Regimens in Enzyme Replacement Therapy of Type I Gaucher Disease
  Blood Cells, Molecules, and Diseases, 2000
• Sandhoff disease in Cyprus: population screening by biochemical and DNA analysis indicates a high frequency of carriers in the Maronite community
  Human Genetics, 2000
• Cathepsin K gene mutations and 1q21 haplotypes in patients with pycnodysostosis in an outbred population
  European Journal of Human Genetics, 2000
• Niemann-Pick disease
  Current Opinion in Hematology, 2000
• Glycoprotein lysosomal storage disorders: α- and β-mannosidosis, fucosidosis and α-N-acetylgalactosaminidase deficiency
  Biochimica et Biophysica Acta (BBA) - Molecular Basis of Disease, 1999
• Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres
  Archives of Disease in Childhood, 1997
• Theearly andlate processing of lysosomal enzymes: Proteolysis and compartmentation
  Cellular and Molecular Life Sciences, 1992
• Sphingolipid activator protein 1 deficiency in metachromatic leucodystrophy with normal arylsulphatase A activity. A clinical, morphological, biochemical and immunological study
  European Journal of Pediatrics, 1991
• REVERSAL OF CLINICAL FEATURES OF HURLER'S DISEASE AND BIOCHEMICAL IMPROVEMENT AFTER TREATMENT BY BONE-MARROW TRANSPLANTATION
  The Lancet, 1981
• Genetic heterogeneity in human neuraminidase deficiency
  Nature, 1980