Developing Angelman syndrome-specific clinician-reported and caregiver-reported measures to support holistic, patient-centered drug development

BACKGROUND: Angelman syndrome (AS) is a rare, heterogenous neurogenetic condition, which significantly impacts the lives of people with AS and their families. Valid and reliable measures reporting key symptoms and functional impairments of AS are required to support development of patient-centered t...

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Autores principales: Connor-Ahmad, Siobhan, Tjeertes, Jorrit, Chladek, Michael, Newton, Louise, Symonds, Tara, Clinch, Susanne, Vincenzi, Brenda, McDougall, Fiona
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10288761/
https://www.ncbi.nlm.nih.gov/pubmed/37349793
http://dx.doi.org/10.1186/s13023-023-02729-y

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