News

Zafgen Suspends Plan to Initiate ZGN-1258 Program as Treatment for PWS

Preclinical studies on the long-term impact of the investigational therapy ZGN-1258, being developed as a potential therapy for Prader-Willi syndrome (PWS), revealed alarming toxicity events and safety issues, Zafgen, the therapy’s developer, has announced. Based on these negative results, the company has changed its plans for ZGN-1258,…

ConSynance Therapeutics Will Participate in Prader-Willi Trial Consortium

ConSynance Therapeutics, a biotechnology company that has developed two new candidate medicines for Prader-Willi syndrome (PWS), announced that it has joined the PWS Clinical Trial Consortium. The PWS Clinical Trial Consortium was established in 2015 to accelerate clinical trials in PWS by promoting multidisciplinary, pre-competitive, and international collaboration among stakeholders…

Tesomet Safe for Teens with PWS; More Data Needed to Show Efficacy

Data from an ongoing Phase 2a clinical trial showed that Tesomet (tesofensine/metoprolol) is safe and well-tolerated by adolescents with Prader-Willi syndrome (PWS). Eight of the nine adolescents included in the trial have enrolled in an open-label extension (OE) study, where they will proceed with Tesomet treatment for an additional…

Vienna to Host RARE2019 Meeting on Rare Diseases

About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…