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...
| Principais autores: | , , , , , , , , |
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| Formato: | Journal article |
| Idioma: | English |
| Publicado em: |
BioMed Central
2018
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