dm+d

Unassigned

New Medicines

Alström syndrome

Information

New molecular entity
Liminal BioSciences
Liminal BioSciences

Development and Regulatory status

Discontinued
Discontinued
Discontinued
Yes
Yes
Apr 22Following the announcement of the PI MAD study results and that fezagepras has potential to show benefit in the treatment of conditions characterised by hyperammonemia, Liminal is to start a single ascending dose (SAD) trial comparing fezagepras with sodium phenylbutyrate. Presume no further development for Alström syndrome [14].

Category

Anti-fibrotic. Regulates inflammation/fibrosis by promoting a type 1 anti-fibrotic cytokine production phenotype in macrophages and T-cells.
Alström syndrome is a very rare autosomal recessive disorder characterised by progressive cone-rod dystrophy leading to blindness, sensorineural hearing loss, childhood obesity associated with hyperinsulinaemia, and type 2 diabetes mellitus [1]. Other common features are dilated cardiomyopathy leading to congestive heart failure, elevated liver enzymes and progressive chronic nephropathy. About 900 affected individuals are currently recorded worldwide [6].
Alström syndrome
Oral