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

Abstract 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...

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Bibliographic Details
Main Authors: Shanat Baig, Vishy Veeranna, Shaun Bolton, Nicola Edwards, Jeremy W. Tomlinson, Konstantinos Manolopoulos, John Moran, Richard P. Steeds, Tarekegn Geberhiwot
Format: Article
Language:English
Published: BMC 2018-11-01
Series:BMC Endocrine Disorders
Subjects:
Alström syndrome
Fibrosis
Insulin resistance
Metabolic syndrome
Diabetes mellitus
Obesity
Online Access:http://link.springer.com/article/10.1186/s12902-018-0315-6

Internet

http://link.springer.com/article/10.1186/s12902-018-0315-6

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