One year of treatment with Soleno Therapeutics’ investigational oral therapy diazoxide choline controlled release, known as DCCR, significantly reduced excessive appetite and disease-related behaviors in Prader-Willi syndrome (PWS) patients, according to top-line data from an extension study of the DESTINY PWS Phase 3 trial. These trajectories were significantly…
People with Prader-Willi syndrome (PWS) have smaller pituitary glands than their typically-developing peers, according to data from a small study. The findings also suggest that altered pituitary size is associated with behavioral abnormalities in PWS. Results were published in the journal Biomarkers in Neuropsychiatry, in the study “…
The mean total annual costs of raising a child with Prader-Willi Syndrome (PWS) — an estimate that takes into account services use, impact on parental income, and care arrangements — is more than AU$57,500 (about $42,500) per child, according to an Australian study of four rare disorders that cause…
An $11.2-million, five-year grant has been awarded by the Centers of Excellence in Genomic Science of the National Institutes of Health (NIH) to uncover high-resolution details of the entire human genome. This project should help better understand the genome’s structure and function, as well as provide important clues about the…
Vision problems such as lazy eye, farsightedness, and strabismus — when the eyes don’t line up in the same direction — are more common in people with Prader-Willi syndrome (PWS) than among their healthy peers, a study shows. These findings, along with data showing that vision problems may negatively affect…
Children and adults with Prader-Willi syndrome (PWS) are not at higher risk of developing a severe COVID-19 infection, according to results from a survey involving more than 500 patients in France. These findings were particularly surprising in terms of the adult population, which typically has several known risk factors for severe…
Pitolisant, a treatment for narcolepsy, should be considered for people with Prader-Willi syndrome (PWS) whose response to psychotropic medications is less than optimal, a case report of a 15-year-old patient suggests. “Pitolisant has the potential to improve many symptoms in patients with Prader-Willi syndrome and it appears to be…
The hunger hormone ghrelin, when measured in infancy, may be a biomarker for predicting the early onset of scoliosis — a sideways curvature of the spine that looks like the letter C or S — in children with Prader-Willi syndrome (PWS), a study suggested. Beginning in early infancy,…
Aardvark Therapeutics has raised $29 million in financing to help support three planned Phase 2 clinical trials of its lead compound, ARD-101, an investigational therapy for Prader-Willi syndrome (PWS) and other disorders. Financing was led by Sorrento Therapeutics, with participation from other investors and support from the…
Growth hormone treatment is safe and helps to increase lean body mass and reducing body fat in adults with Prader-Willi syndrome (PWS), according to an analysis of published studies. Findings also suggest that such improvements in body composition might lower the risk of cardiovascular problems in adults with the…
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