Bilaketaren emaitzak - Weinreb, Neal

Patients with type 1 Gaucher disease in South Florida, USA: demographics, genotypes, disease severity and treatment outcomes nork Orenstein, Marissa, Barbouth, Deborah, Bodamer, Olaf A, Weinreb, Neal J

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Gaucher disease: Resetting the clinical and scientific agenda nork Mistry, Pramod K., Weinreb, Neal J., Brady, Roscoe O., Grabowski, Gregory A.

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MGUS, lymphoplasmacytic malignancies, and Gaucher disease: the significance of the clinical association nork Weinreb, Neal J., Mistry, Pramod K., Rosenbloom, Barry E., Dhodapkar, Madhav V.

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The International Collaborative Gaucher Group GRAF (Gaucher Risk Assessment for Fracture) score: a composite risk score for assessing adult fracture risk in imiglucerase-treated Ga... nork Deegan, Patrick, Khan, Aneal, Camelo, José Simon, Batista, Julie L., Weinreb, Neal

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GBA1 mutations: Prospects for Exosomal Biomarkers in α-Synuclein Pathologies nork Johnson, Parker, Weinreb, Neal J., Cloyd, James, Tuite, Paul J, Kartha, Reena V.

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Modeling potential interactions between oral Gaucher disease treatment and investigational COVID-19 therapies nork Sahasrabudhe, Siddhee A., Al-Kofahi, Mahmoud, Cloyd, James C., Weinreb, Neal, Kartha, Reena V.

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Timing of initiation of enzyme replacement therapy after diagnosis of type 1 Gaucher disease: effect on incidence of avascular necrosis nork Mistry, Pramod K, Deegan, Patrick, Vellodi, Ashok, Cole, J Alexander, Yeh, Michael, Weinreb, Neal J

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A new framework for evaluating the health impacts of treatment for Gaucher disease type 1 nork Ganz, Michael L., Stern, Sean, Ward, Alex, Nalysnyk, Luba, Selzer, Martin, Hamed, Alaa, Weinreb, Neal

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Gaucher Disease: Progress and Ongoing Challenges nork Mistry, Pramod K., Lopez, Grisel, Schiffmann, Raphael, Barton, Norman W., Weinreb, Neal J., Sidransky, Ellen

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Osteopenia in Gaucher disease develops early in life: Response to imiglucerase enzyme therapy in children, adolescents and adults nork Mistry, Pramod K., Weinreb, Neal J., Kaplan, Paige, Cole, J. Alexander, Gwosdow, Andrea R., Hangartner, Thomas

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Reducing selection bias in case-control studies from rare disease registries nork Cole, J Alexander, Taylor, John S, Hangartner, Thomas N, Weinreb, Neal J, Mistry, Pramod K, Khan, Aneal

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Enzyme replacement and substrate reduction therapy for Gaucher disease nork Shemesh, Elad, Deroma, Laura, Bembi, Bruno, Deegan, Patrick, Hollak, Carla, Weinreb, Neal J, Cox, Timothy M

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A multicenter open-label treatment protocol (HGT-GCB-058) of velaglucerase alfa enzyme replacement therapy in patients with Gaucher disease type 1: safety and tolerability nork Pastores, Gregory M., Rosenbloom, Barry, Weinreb, Neal, Goker-Alpan, Ozlem, Grabowski, Gregory, Cohn, Gabriel M., Zahrieh, David

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Life expectancy in Gaucher disease type 1 nork Weinreb, Neal J., Deegan, Patrick, Kacena, Katherine A., Mistry, Pramod, Pastores, Gregory M., Velentgas, Priscilla, vom Dahl, Stephan

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Evaluation of disease burden and response to treatment in adults with type 1 gaucher disease using a validated disease severity scoring system (DS3) nork Weinreb, Neal J., Finegold, David N., Feingold, Eleanor, Zeng, Zhen, Rosenbloom, Barry E., Shankar, Suma P., Amato, Dominick

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Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment nork Weinreb, Neal J., Goldblatt, Jack, Villalobos, Jacobo, Charrow, Joel, Cole, J. Alexander, Kerstenetzky, Marcelo, vom Dahl, Stephan, Hollak, Carla

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Erratum to: Long-term clinical outcomes in type 1 Gaucher disease following 10 years of imiglucerase treatment nork Weinreb, Neal J., Goldblatt, Jack, Villalobos, Jacobo, Charrow, Joel, Cole, J. Alexander, Kerstenetzky, Marcelo, vom Dahl, Stephan, Hollak, Carla

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Effect and Tolerability of Agalsidase Alfa in Patients with Fabry Disease Who Were Treatment Naïve or Formerly Treated with Agalsidase Beta or Agalsidase Alfa nork Goker-Alpan, Ozlem, Nedd, Khan, Shankar, Suma P., Lien, Yeong-Hau H., Weinreb, Neal, Wijatyk, Anna, Chang, Peter, Martin, Rick

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Reduction of Plasma Globotriaosylsphingosine Levels After Switching from Agalsidase Alfa to Agalsidase Beta as Enzyme Replacement Therapy for Fabry Disease nork Goker-Alpan, Ozlem, Gambello, Michael J., Maegawa, Gustavo H. B., Nedd, Khan J., Gruskin, Daniel J., Blankstein, Larry, Weinreb, Neal J.

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Gaucher Disease in Bone: From Pathophysiology to Practice nork Hughes, Derralynn, Mikosch, Peter, Belmatoug, Nadia, Carubbi, Francesca, Cox, TimothyM, Goker‐Alpan, Ozlem, Kindmark, Andreas, Mistry, PramodK, Poll, Ludger, Weinreb, Neal, Deegan, Patrick

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