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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...
| Autores principales: | , , , , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
John Wiley & Sons, Inc.
2022
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| Materias: | |
| 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 |