Alstrom Syndrome Treatment News and Symptoms Information

What is Alström Syndrome?

(Symptoms, Causes & Treatment)

Alstrom Syndrome is a rare genetic disease caused by mutations in the ALMS1 gene, which can affect many different parts of the body. According to the U.S. National Library of Medicine, Alström syndrome is characterized by a progressive loss of vision and hearing, a form of heart disease that enlarges and weakens the heart muscle (dilated cardiomyopathy), obesity, type 2 diabetes mellitus (the most common form of diabetes), and short stature. This disorder can also cause serious or life-threatening medical problems involving the liver, kidneys, bladder, and lungs. Some individuals with Alström syndrome have a skin condition called acanthosis nigricans, which causes the skin in body folds and creases to become thick, dark, and velvety.

See below for updated news and information regarding Alstrom Syndrome including new medical research, treatment options and advancements. 

Latest Alstrom Syndrome Treatment News and Research

European Commission grants orphan drug designation to ProMetic’s

PR Newswire (press release)Jan 18, 2017
European Commission grants orphan drug designation to ProMetic’s PBI-4050 drug for the treatment of Alström Syndrome …

Prometic’s PBI-4050 gets promising innovative medicine …

pharmabiz.comJan 25, 2017
We are pleased to see the MHRA agree that the data generated so far with PBI-4050 in the Alström Syndrome phase 2 clinical trial …

PBI-4050 Reduces Liver Fibrosis in Alström Syndrome

Rare Disease ReportOct 12, 2016
The ongoing Alström syndrome phase 2 clinical trial is an open label, single arm and single center study investigating the safety, tolerability …

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