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

Bingaman, Amanda; Waggoner, Christine; Andrews, Sara M; Pangonis, Diana; Trad, Marie; Giugliani, Roberto; Giorgino, Ruben; Jarnes, Jeanine; Vakili, Rojan; Ballard, Victoria; Peay, Holly L.

Afiliação

Bingaman A; RTI International, Research Triangle, North Carolina, United States.
Waggoner C; Cure GM1 Foundation, Albany, California, United States.
Andrews SM; RTI International, Research Triangle, North Carolina, United States.
Pangonis D; National Tay-Sachs & Allied Diseases Association (NTSAD), Brighton, Massachusetts, United States.
Trad M; Lysogene, Neuilly-sur-Seine, France.
Giugliani R; PPGBM UFRGS, DASA Genomics and Casa dos Raros, Porto Alegre, Rio Grande do Sul, Brazil.
Giorgino R; Azafaros, Leiden, Zuid-Holland, The Netherlands.
Jarnes J; Department of Pediatrics, University of Minnesota, Minneapolis, Minnesota, United States.
Vakili R; Cure GM1 Foundation Member, Albany, CA, USA.
Ballard V; Passage Bio, Inc., Philadelphia, Pennsylvania, United States.
Peay HL; RTI International, Research Triangle, North Carolina, United States.

Am J Med Genet A ; 191(2): 408-423, 2023 02.

Article em En | MEDLINE | ID: mdl-36541412

Assuntos

Cuidadores; Gangliosidose GM1; Criança; Humanos; Qualidade de Vida; Gangliosídeo G(M1); Pais; Doenças Raras

Palavras-chave

GM1; burden; caregivers; patient-focused drug development; treatment priorities

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Gangliosidose GM1 / Cuidadores Tipo de estudo: Diagnostic_studies / Qualitative_research Limite: Child / Humans Idioma: En Ano de publicação: 2023 Tipo de documento: Article

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