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International Journal of Molecular Sciences|May 13, 2026
Glucosylsphingosine (Lyso-Gb1) Dynamics in Untreated States in Gaucher DiseaseTama Dinur, Peter Bauer, Sabine Schroeder, et al.
Blood Advances|February 5, 2024
Management of acute breakthrough hemolysis with intensive pegcetacoplan dosing in patients with PNHMorag Griffin, Richard J Kelly, Jens Panse, et al.
American Journal of Hematology|March 7, 2026
Real-World Effectiveness and Safety of Ravulizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria: Evidence From the International PNH RegistryAlexander Röth, Christopher J Patriquin, Jeff Szer, et al.
Molecular Biology International|January 16, 2013
Prevention of lysosomal storage diseases and derivation of mutant stem cell lines by preimplantation genetic diagnosisGheona Altarescu, Rachel Beeri, Rachel Eiges, et al.
The Journal of Obstetrics and Gynaecology Research|March 12, 2014
Outcome of pregnancies in women receiving velaglucerase alfa for Gaucher diseaseDeborah Elstein, Derralynn Hughes, Ozlem Goker-Alpan, et al.
Journal of Clinical Medicine|May 25, 2024
Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) RegistryPatrick Deegan, Heather Lau, Deborah Elstein, et al.
European Journal of Haematology|April 26, 2025
Anchored Indirect Treatment Comparison Finds Comparable Effects of Pegcetacoplan and Iptacopan in Paroxysmal Nocturnal HaemoglobinuriaRegis Peffault de Latour, Austin Kulasekararaj, David Dingli, et al.
Blood Advances|April 9, 2024
Hemolysis events in the phase 3 PEGASUS study of pegcetacoplan in patients with paroxysmal nocturnal hemoglobinuriaRégis Peffault de Latour, Morag Griffin, Richard J Kelly, et al.
Haematologica|February 1, 2020
Accuracy of chitotriosidase activity and CCL18 concentration in assessing type I Gaucher disease severity. A systematic review with meta-analysis of individual participant dataTatiana Raskovalova, Patrick B Deegan, Pramod K Mistry, et al.
Neurology. Genetics|April 29, 2016
Clinical course and prognosis in patients with Gaucher disease and parkinsonismGrisel Lopez, Jenny Kim, Edythe Wiggs, et al.
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