Keyword Connections

enzyme replacement therapy

Journals

2

1 Genetics in medicine : official journal of the American College of Medical Genetics

2 jimd reports

Research Groups

0

No Research Group Connected

Bibliographies

12

1 The clinical profiles of female patients with Fabry disease in Latin America: A Fabry Registry analysis of natural history data from 169 patients based on enzyme replacement therapy status.

2 Higher dosing of alglucosidase alfa improves outcomes in children with Pompe disease: a clinical study and review of the literature.

3 a pilot study on using rapamycin-carrying synthetic vaccine particles (svp) in conjunction with enzyme replacement therapy to induce immune tolerance in pompe disease

4 control of proteinuria with increased doses of agalsidase alfa in a patient with fabry disease with atypical genotype–phenotype expression

5 lipid profile in adult patients with fabry disease - ten-year follow up

6 successful reduction of high-sustained anti-idursulfase antibody titers by immune modulation therapy in a patient with severe mucopolysaccharidosis type ii

7 pompe disease: from pathophysiology to therapy and back again

8 home infusion program with enzyme replacement therapy for fabry disease: the experience of a large italian collaborative group

9 risks of long-term port use in enzyme replacement therapy for lysosomal storage disorders

10 quality of life assessment after pancreatic enzyme replacement therapy in chronic pancreatitis

11 mucopolysaccharidosis type vi on enzyme replacement therapy since infancy: six years follow-up of four children

12 safety and efficacy of enzyme replacement therapy in the nephropathy of fabry disease