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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...

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Detalles Bibliográficos
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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2023
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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