Dados do Trabalho
Título
REAL WORLD SAFETY AND EXPLORATORY EFFICACY OF GENE THERAPY FOR PATIENTS WITH 5Q-SPINAL MUSCULAR ATROPHY IN A BRAZILIAN COHORT
Palavras-chave
Spinal Muscular Atrophy, Gene Therapy, Adeno-associated Virus serotype 9 (AAV9), Survival Motor Neuron 1 gene (SMN1)
Área
Doenças Neuromusculares
Autores
Rodrigo Holanda Mendonça, Adriana Banzatto Ortega, Ciro Matsui Jr, Vanessa Van Der Linden, Marcelo Kerstenetzky, Luis Fernando Grossklauss, Elizabeth Lemos S Lucas, Graziela Jorge Polido, Edmar Zanoteli