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

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

<strong>Background</strong> 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 resistanc...

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Dades bibliogràfiques
Autors principals:	Baig, S, Veeranna, V, Bolton, S, Edwards, N, Tomlinson, J, Manolopoulos, K, Moran, J, Steeds, R, Geberhiwot, T
Format:	Journal article
Idioma:	English
Publicat:	BioMed Central 2018

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