Midline defects in FG syndrome: Does tethered spinal cord contribute to the phenotype?
65 Fabry disease female heterozygotes are not just “carriers”, but have a significant burden of disease and impaired quality of life
147. Early treatment initiation for MPS reduces severity of brain imaging abnormalities
Intra-articular Enzyme Replacement Therapy with Recombinant Human Iduronidase is Safe and Well Tolerated in the Canine Model of Mucopolysaccharidosis Type I
Carotid Intima-Media thickness is increased in patients with mucopolysaccharidoses
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