Early therapeutic intervention in females with Fabry disease?
Author:
Publisher
Wiley
Subject
General Medicine,Pediatrics, Perinatology and Child Health
Link
http://onlinelibrary.wiley.com/wol1/doi/10.1111/j.1651-2227.2008.00649.x/fullpdf
Reference54 articles.
1. Fabry's Disease: Alpha-Galactosidase Deficiency
2. Fabry disease defined: baseline clinical manifestations of 366 patients in the Fabry Outcome Survey
3. Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 98 hemizygous males
4. Anderson-Fabry disease: clinical manifestations and impact of disease in a cohort of 60 obligate carrier females
5. A Phase 1/2 Clinical Trial of Enzyme Replacement in Fabry Disease: Pharmacokinetic, Substrate Clearance, and Safety Studies
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1. Evaluation of Beta 2-Microglobulin, Cystatin C, and Lipocalin-2 as Renal Biomarkers for Patients with Fabry Disease;Nephron;2019
2. Oral Migalastat HCl Leads to Greater Systemic Exposure and Tissue Levels of Active α-Galactosidase A in Fabry Patients when Co-Administered with Infused Agalsidase;PLOS ONE;2015-08-07
3. The attenuated/late onset lysosomal storage disorders: Therapeutic goals and indications for enzyme replacement treatment in Gaucher and Fabry disease;Best Practice & Research Clinical Endocrinology & Metabolism;2015-03
4. Pharmacological Chaperones as Therapeutics for Lysosomal Storage Diseases;Journal of Medicinal Chemistry;2013-03-11
5. Females and children with Anderson–Fabry disease: diagnosis, monitoring, benefits of enzyme replacement therapy (ERT) and considerations on timing of starting ERT;Expert Opinion on Orphan Drugs;2013-03-06
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