Navegação Ciências da Saúde por Assunto "Treatment priorities"

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    • GM1-gangliosidosis : the caregivers' assessments of symptom impact and most important symptoms to treat 

      Bingaman, Amanda; Waggoner, Christine; Andrews, Sara M.; Pangonis, Diana; Trad, Marie; Giugliani, Roberto; Giorgino, Ruben; Jarnes, Jeanine; Vakili, Rojan; Ballard, Victoria; Peay, Holly L. (2023) [Artigo de periódico]
      GM1-gangliosidosis (GM1) is a rare neurodegenerative disorder leading to early mortality and causing progressive decline of physical skills and cerebral functioning. No approved treatment for GM1 exists. In this study-the ...