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Quality of life improvements following one year of setmelanotide in children and adult patients with Bardet–Biedl syndrome: phase 3 trial results
BACKGROUND: Bardet–Biedl syndrome is a rare genetic disease associated with hyperphagia and early-onset, severe obesity. There is limited evidence on how hyperphagia and obesity affect health-related quality of life in patients with Bardet–Biedl syndrome, and on how management of these symptoms may...
Autores principales: | Forsythe, Elizabeth, Haws, Robert M., Argente, Jesús, Beales, Philip, Martos-Moreno, Gabriel Á., Dollfus, Hélène, Chirila, Costel, Gnanasakthy, Ari, Buckley, Brieana C., Mallya, Usha G., Clément, Karine, Haqq, Andrea M. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9841937/ https://www.ncbi.nlm.nih.gov/pubmed/36647077 http://dx.doi.org/10.1186/s13023-022-02602-4 |
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