Biodistribution and pharmacodynamics of recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations. (65/130)

Molecular analysis of mucopolysaccharidosis type I in Tunisia: identification of novel mutation and eight Novel polymorphisms. (66/130)

Clinical manifestations and treatment of mucopolysaccharidosis type I patients in Latin America as compared with the rest of the world. (67/130)

Mucopolysaccharidosis type I: molecular characteristics of two novel alpha-L-iduronidase mutations in Tunisian patients. (68/130)

Effect of one year of cryopreservation on the activity of lysosomal hydrolases from EBV-transformed lymphocytes. (69/130)

Synthetic zinc finger nuclease design and rapid assembly. (70/130)

Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase. (71/130)

Enzyme replacement therapy and/or hematopoietic stem cell transplantation at diagnosis in patients with mucopolysaccharidosis type I: results of a European consensus procedure. (72/130)

• 1
• 2
• 3
• 4
• 5
• 6
• 7
• 8
• 9
• 10