Treatment with PBI-4050 in patients with Alström syndrome: study protocol for a phase 2, single-Centre, single-arm, open-label trial

BACKGROUND: Alström syndrome (ALMS) is a very rare autosomal recessive monogenic disorder caused by a mutation in the ALMS1 gene and characterised by childhood onset obesity, dyslipidaemia, advanced non-alcoholic fatty liver disease, diabetes and extreme insulin resistance. There is evidence of mult...

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Detalles Bibliográficos
Autores principales: Baig, Shanat, Veeranna, Vishy, Bolton, Shaun, Edwards, Nicola, Tomlinson, Jeremy W., Manolopoulos, Konstantinos, Moran, John, Steeds, Richard P., Geberhiwot, Tarekegn
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Study Protocol
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6258144/
https://www.ncbi.nlm.nih.gov/pubmed/30477455
http://dx.doi.org/10.1186/s12902-018-0315-6

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