GM1‐gangliosidosis: The caregivers' assessments of symptom impact and most important symptoms to treat

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 first to explore priorities of parents of subjects with pediatric onset forms...

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Detalles Bibliográficos
Autores principales: Bingaman, Amanda, Waggoner, Christine, Andrews, Sara M., Pangonis, Diana, Trad, Marie, Giugliani, Roberto, Giorgino, Ruben, Jarnes, Jeanine, Vakili, Rojan, Ballard, Victoria, Peay, Holly L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley & Sons, Inc. 2022
Materias:
Original Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10107815/
https://www.ncbi.nlm.nih.gov/pubmed/36541412
http://dx.doi.org/10.1002/ajmg.a.63038

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10107815/
https://www.ncbi.nlm.nih.gov/pubmed/36541412
http://dx.doi.org/10.1002/ajmg.a.63038

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