15 results on '"Deegan, Patrick B."'
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2. Lyso-Gb1 as a biomarker of the real-world situation in Gaucher disease: Comparative data from the Gaucher Outcome Survey (GOS) in ERT-treated and untreated patients
3. Long-term safety and efficacy of pegunigalsidase alfa administered every 4 weeks in patients with Fabry disease: Two-year interim results from the ongoing phase 3 BRIGHT51 open-label extension study
4. Venglustat, an orally administered glucosylceramide synthase inhibitor: Assessment over 3 years in adult males with classic Fabry disease in an open-label phase 2 study and its extension study
5. A study to evaluate the effect of venglustat on neuropathic and abdominal pain in symptomatic adult patients with Fabry disease
6. Risk factors for fracture in imiglucerase-treated Gaucher disease type 1 patients in the ICGG Gaucher Registry
7. A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase
8. Sebelipase alfa over 52weeks reduces serum transaminases, liver volume and improves serum lipids in patients with lysosomal acid lipase deficiency
9. Novel mutations of ABCA1 transporter in patients with Tangier disease and familial HDL deficiency
10. Response of women with Fabry disease to enzyme replacement therapy: Comparison with men, using data from FOS—the Fabry Outcome Survey
11. A novel tool for mapping disease severity and outcomes in patients with Gaucher disease utilising the therapeutic goals
12. Potential biomarkers of osteonecrosis in Gaucher disease
13. Foreword
14. Clinical evaluation of chemokine and enzymatic biomarkers of Gaucher disease
15. Prognostic value of troponin T in hemodialysis patients is independent of comorbidity
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