Article
Skeletal involvement in Gaucher disease: An observational multicenter study of prognostic factors in the Argentine Gaucher disease patients.
American journal of hematology - 1 Oct 2016
Drelichman Guillermo, Fernández Escobar Nicolás, Basack Nora, Aversa Luis, Larroude María Silvia, Aguilar Gabriela, Szlago Marina, Schenone Andrea, Fynn Alcyra, Cuello María Fernanda, Aznar Marcela, Fernández Ramiro, Ruiz Alba, Reichel Paola, Guelbert Norberto, Robledo Hugo, Watman Nora, Bolesina Moira, Elena Graciela, Veber S Ernesto, Pujal Graciela, Galván Graciela, Chain Juan José, Arizo Adriana, Bietti Julieta, Bar Daniel, Dragosky Marta, Marquez Marisa, Feldman Leonardo, Muller Katja, Zirone Sandra, Buchovsky Greogorio, Lanza Victoria, Sanabria Alba, Fernández Ignacio, Jaureguiberry Rossana, Contte Marcelo, Barbieri María Angie, Maro Alejandra, Zárate Graciela, Fernández Gabriel, Rapetti María Cristina, Donato Hugo, Degano Adriana, Kantor Gustavo, Albina Roberto, Á Lvarez Bollea María, Brun María, Bacciedoni Viviana, Del Río Francis, Soberón Bárbara, Boido Nazario, Schweri Maya, Borchichi Sandra, Welsh Victoria, Corrales Marcela, Cedola Alejandra, Carvani Analía, Diez Blanca, Richard Lucía, Baduel Ccecilia, Nuñez Gabriela, Colimodio Rubén, Barazzutti Lucía, Medici Hugo, Meschengieser Susana, Damiani Germán, Nucifora María, Girardi Beatriz, Gómez Sergio, Papucci Maura, Verón David, Quiroga Luis, Carro Gustavo, De Ambrosio Patricia, Ferro José, Pujol Marcelo, Castella Cristina Cabral, Franco Liliana, Nisnovich Gisela, Veloso María, Pacheco Isabel, Savarino Mario, Marino Andrés, Saavedra José Luis
Abstract excerpt
Patients with Gaucher type 1 (GD1) throughout Argentina were enrolled in the Argentine bone project to evaluate bone disease and its determinants. We focused on presence and predictors of bone lesions (BL) and their relationship to therapeutic goals (TG) with timing and dose of enzyme replacement therapy (ERT). A total of 124 patients on ERT were enrolled in a multi-center study. All six TG were achieved by 82%...
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