A compound that could mimic ghrelin hormone activity and trigger its receptor signals could hold therapeutic potential to improve survival of patients with Prader-Willi syndrome (PWS) in early infancy, a mouse study shows. The study, “Ghrelin Receptor Agonist Rescues Excess…
Millendo Therapeutics has launched a Phase 2b/3 clinical trial investigating the safety and efficacy of livoletide (AZP-531) in patients with Prader-Willi syndrome (PWS), the company announced. The rare genetic disease is characterized by hyperphagia (excessive eating) and obesity, caused by abnormal hormonal levels. Livoletide is designed to reduce…
The Data Safety Monitoring Board recommended the continuation of Soleno Therapeutics’ Phase 3 clinical trial evaluating the effects of diazoxide choline controlled release (DCCR) treatment in children and adults with Prader-Willi syndrome (PWS), the company announced. Hyperphagia (excessive eating) is a classic PWS symptom caused by hormonal or…
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,…
Madeline Collin, a 24-year-old activist with Gaucher disease, worries that patients like her will suffer deeply if Britain leaves the European Union (EU), as scheduled, at the end of this month. Collin is an expert on the subject. For her University of Bathdissertation, she analyzed Brexit’s long-term impact…
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…
With each new advance in medicine comes ethical dilemmas, from fertility treatments and newborn screening, to vaccinations, gene therapies and euthanasia. But rare diseases and the expensive therapies needed to treat them — particularly in an age of scarce economic resources — almost always entail “tragic choices,” warned Avraham Steinberg,…
Rare diseases affect about 30 million Americans — roughly the same number as those with type 2 diabetes. Yet only 5 percent of the estimated 7,000 rare diseases known to science have cures or treatments approved by the U.S. Food and Drug Administration (FDA). Raising awareness of those illnesses and highlighting…
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…
The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…
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