Need leads to change: Transition to home infusion in Pompe disease in Brazil in the COVID-19 pandemic

por Horovitz, Dafne Dain Gandelman, Barth, Anneliese Lopes, Alves, Welton Correia, Llerena, Juan Clinton
Publicado 2021

Enzyme replacement therapy interruption in mucopolysaccharidosis type IVA patients and its impact in different clinical outcomes

por Politei, Juan, Porras‐Hurtado, Gloria Liliana, Guelbert, Norberto, Fainboim, Alejandro, Horovitz, Dafne Dain Gandelman, Satizábal, José María
Publicado 2021

Genetics and genomics in Brazil: a promising future

por Passos-Bueno, Maria Rita, Bertola, Debora, Horovitz, Dafne Dain Gandelman, de Faria Ferraz, Victor Evangelista, Brito, Luciano Abreu
Publicado 2014

Otolaryngologists and the Early Diagnosis of Mucopolysaccharidoses: A Cross-Sectional Study

por Torres, Danielle de Araujo, Barth, Anneliese Lopes, Valente, Mariana Pires de Mello, de Mello, Paulo Pires, Horovitz, Dafne Dain Gandelman
Publicado 2019

Neurological Phenotypes of IRF2BPL Gene Variants: A Report of Four Novel Variants

por Horovitz, Dafne Dain Gandelman, de Faria Domingues de Lima, Maria Angelica, Pires, Lais de Carvalho, Campos Araujo, Abelardo de Queiroz, Vargas, Fernando Regla
Publicado 2023

Respiratory manifestations in late-onset Pompe disease: a case series conducted in Brazil

por Sixel, Bruna de Souza, da Silva, Luanda Dias, Cavalcanti, Nicolette Celani, Penque, Glória Maria Cardoso de Andrade, Lisboa, Sandra, Horovitz, Dafne Dain Gandelman, Llerena, Juan Clinton
Publicado 2017

Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series

por Horovitz, Dafne Dain Gandelman, Acosta, Angelina X., Giugliani, Roberto, Hlavatá, Anna, Hlavatá, Katarína, Tchan, Michel C., Lopes Barth, Anneliese, Cardoso, Laercio, Embiruçu de Araújo Leão, Emília Katiane, Esposito, Ana Carolina, Kyosen, Sandra Obikawa, De Souza, Carolina Fischinger Moura, Martins, Ana Maria
Publicado 2016

Enzyme replacement therapy for Mucopolysaccharidosis Type I among patients followed within the MPS Brazil Network

por Dornelles, Alícia Dorneles, de Camargo Pinto, Louise Lapagesse, de Paula, Ana Carolina, Steiner, Carlos Eduardo, Lourenço, Charles Marques, Kim, Chong Ae, Horovitz, Dafne Dain Gandelman, Ribeiro, Erlane Marques, Valadares, Eugênia Ribeiro, Goulart, Isabela, Neves de Souza, Isabel C., da Costa Neri, João Ivanildo, Santana-da-Silva, Luiz Carlos, Silva, Luiz Roberto, Ribeiro, Márcia, de Oliveira Sobrinho, Ruy Pires, Giugliani, Roberto, Schwartz, Ida Vanessa Doederlein
Publicado 2014

Mucopolysaccharidosis VII in Brazil: natural history and clinical findings

por Giugliani, Roberto, Barth, Anneliese Lopes, Dumas, Melissa Rossi Calvão, da Silva Franco, José Francisco, de Rosso Giuliani, Liane, Grangeiro, Carlos Henrique Paiva, Horovitz, Dafne Dain Gandelman, Kim, Chong Ae, de Araújo Leão, Emilia Katiane Embiruçu, de Medeiros, Paula Frassinetti Vasconcelos, Miguel, Diego Santana Chaves Geraldo, Moreira, Maria Espírito Santo Almeida, dos Santos, Helena Maria Guimarães Pimentel, da Silva, Luiz Carlos Santana, da Silva, Luiz Roberto, de Souza, Isabel Neves, Nalin, Tatiele, Garcia, Daniel
Publicado 2021