Sort   by:  
 Page size 
UnknownLong-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment.
Not available
S-EPMC3648688 | biostudies-literature
UnknownReal-world effectiveness of eliglustat in treatment-naive and switch patients enrolled in the International Collaborative Gaucher Group Gaucher Registry.
Not available
S-EPMC7497238 | biostudies-literature
UnknownA rapid LC-MS/MS assay for detection and monitoring of underivatized branched-chain amino acids in maple syrup urine disease.
Not available
S-EPMC9120951 | biostudies-literature
UnknownAntiproteinuric therapy and Fabry nephropathy: factors associated with preserved kidney function during agalsidase-beta therapy.
Not available
S-EPMC4717450 | biostudies-literature
UnknownTranscript analysis for variant classification resolution in a child with primary ciliary dyskinesia.
Not available
S-EPMC7903884 | biostudies-literature
Unknownβ-Glucocerebrosidase Modulators Promote Dimerization of β-Glucocerebrosidase and Reveal an Allosteric Binding Site.
Not available
S-EPMC6098685 | biostudies-literature
UnknownTen-year outcome of enzyme replacement therapy with agalsidase beta in patients with Fabry disease.
Not available
S-EPMC4413801 | biostudies-literature
UnknownSchaaf-Yang syndrome overview: Report of 78 individuals.
Not available
S-EPMC6585857 | biostudies-literature
UnknownTransformation in pretreatment manifestations of Gaucher disease type 1 during two decades of alglucerase/imiglucerase enzyme replacement therapy in the International Collaborative Gaucher Group (ICGG) Gaucher Registry.
Not available
S-EPMC5600096 | biostudies-literature
UnknownTime to treatment benefit for adult patients with Fabry disease receiving agalsidase β: data from the Fabry Registry.
Not available
S-EPMC4941144 | biostudies-literature
Sort   by:  
 Page size