Research Findings Provide Insights – The Alstrom Syndrome Journey

In the course of their effort to advance trial participation and to ultimately gain approval for the anti-hyperphagia drug Setmelanotide, Rhythm Pharmaceutical, with the cooperation of Alstrom Syndrome International, generated the following report to aid those interested in or needing to understand the lived experience of those impacted by Alstrom Syndrome. Ultimately, the FDA did not approve the use of Setmelanotide for treating Alstrom patients in the USA at this time. However, this situation and others like it spurred ASI on to seek and secure permission to participate in the FDA’s Externally-Led (ASI) Patient Focused Drug Development Initiative, an effort beautifully executed by mostly patients and parents with an all-day presentation, broadcast globally, this past September. AN UNQUALIFIED SUCCESS! View Report

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