Classic and Atypical Late Infantile Neuronal Ceroid Lipofuscinosis in Latin America: Clinical and Genetic Aspects, and Treatment Outcome with Cerliponase Alfa

Guelbert, Norberto; Espitia Segura, Oscar Mauricio; Amoretti, Carolina; Arteaga Arteaga, Angélica; Atanacio, Nora Graciela; Bazan Natacha, Sabrina; Carvalho, Ellaine Doris Fernandes; Carvalho de Andrade, Maria Denise Fernandes; Denzler, Inés María; Durand, Consuelo; Ribeiro, Erlane; Giugni, Juan Carlos; González, Gabriel; González Moron, Dolores; Guelbert, Guillermo; Hernández Rodriguez, Zulma Janneth; Embiruçu Emilia, Katiane; Kauffman, Marcelo Andrés; Mancilla, Nury Isabel; Marcon, Laureano; Marques Pereira, Alessandra; Fischinger Moura de Souza, Carolina; Muñoz, Victor Adrián; Naranjo Flórez, Ricardo Andrés; Pessoa, André Luiz; Ruiz, María Victoria; Solano Villareal, Martha Luz; Spécola, Norma; Tavera, Lina Marcela; Tello, Javiera; Troncoso Schifferli, Mónica; Ugrina, Sonia; Vaccarezza, María Magdalena; Vergara, Diane; Villanueva, María Mercedes

doi:10.1016/j.ymgmr.2024.101060

Molecular Genetics and Metabolism Reports

2024

DOI: 10.1016/j.ymgmr.2024.101060

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Classic and Atypical Late Infantile Neuronal Ceroid Lipofuscinosis in Latin America: Clinical and Genetic Aspects, and Treatment Outcome with Cerliponase Alfa

Norberto Guelbert,

Oscar Mauricio Espitia Segura,

Carolina Amoretti

et al.

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Classic and Atypical Late Infantile Neuronal Ceroid Lipofuscinosis in Latin America: Clinical and Genetic Aspects, and Treatment Outcome with Cerliponase Alfa

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