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收藏The Gaucher Disease Outcome Survey: Description of the population in an ongoing international observational disease registry
Elstein, Deborah; Schwartz, Ida; Deegan, Patrick; Lau, Heather; Goker-Alpan, Ozlem; Lukina, Elena A.; Bembi, Bruno; Belmatoug, Nadia; Fernandez Sasso, Diego; Giraldo, Pilar; Fabien, Vincent; Dinh, Quinn; Zimran, Ari
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收藏Proof-of-principle rapid noninvasive prenatal diagnosis of autosomal recessive founder mutations
Zeevi, David A.; Altarescu, Gheona; Weinberg-Shukron, Ariella; Zandeh, Fouad; Dinur, Tama; Chicco, Gaya; Herskovitz, Yair; Renbaum, Paul; Elstein, Deborah; Levy-Lahad, Ephrat; Rolfs, Arndt; Zimran, Ari
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收藏Safety and efficacy results of switch from imiglucerase to velaglucerase alfa treatment in patients with type 1 Gaucher disease
Elstein, Deborah; Mehta, Atul; Hughes, Derralynn A.; Giraldo, Pilar; Charrow, Joel; Smith, Laurie; Shankar, Suma P.; Hangartner, Thomas N.; Kunes, Yune; Wang, Nan; Crombez, Eric; Zimran, Ari
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收藏Velaglucerase alfa (VPRIV) enzyme replacement therapy in patients with Gaucher disease: Long-term data from phase III clinical trials
Hughes, Derralynn A.; Gonzalez, Derlis E.; Lukina, Elena A.; Mehta, Atul; Kabra, Madhulika; Elstein, Deborah; Kisinovsky, Isaac; Giraldo, Pilar; Bavdekar, Ashish; Hangartner, Thomas N.; Wang, Nan; Crombez, Eric; Zimran, Ari
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收藏Effect of cyclic, low dose pyrimethamine treatment in patients with Late Onset Tay Sachs: an open label, extended pilot study
Osher, Etty; Fattal-Valevski, Aviva; Sagie, Liora; Urshanski, Nataly; Sagiv, Nadav; Peleg, Leah; Lerman-Sagie, Tally; Zimran, Ari; Elstein, Deborah; Navon, Ruth; Valevski, Avi; Stern, Naftali
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收藏Gastrointestinal manifestations of immune dysregulation and Gaucher disease: Mesenteric lymphadenopathy and enteropathy with profound T cell defects
Limgala, Renuka P.; Abrahamov, Aya; Hadas-Halpern, Irith; Ryherd, Mark; Austin, Lauren; Plassmeyer, Matthew; Brown, Margaret; Alpan, Oral; Elstein, Deborah; Zimran, Ari; Goker-Alpan, Ozlem
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