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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…
Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending the event in person in San Diego. “Here you’ll have the opportunity to connect and engage with others…
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,…
Registration is now open for the 2021 Rare Diseases and Orphan Products Breakthrough Summit, which will be held virtually Oct. 18–19. The event, also known as the National Organization for Rare Disorders (NORD) Summit, brings the rare disease community together to network and discuss developments in treatments and research…
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…
More than 600 people participated in the 10th annual Rare Disease Week on Capitol Hill 2021, held virtually July 14–22, to advocate for the rare disease community. Hosted by the EveryLife Foundation’s Rare Disease Legislative Advocates (RDLA) program, the event brings together community members from across the U.S. to…
Radius Health plans to initiate a Phase 2/3 study to evaluate the safety and efficacy of its investigational therapy RAD011, a lab-made oral cannabidiol therapy to treat excessive appetite, or hyperphagia, in people with Prader-Willi syndrome (PWS). The study, called SCOUT-015, is expected to begin by the…
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