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A case of acute kidney injury in a woman with Prader-Willi syndrome (PWS) and intellectual disability was attributed in a recent report to the use of multiple medications. The case shed lights on how vulnerable people with an intellectual disability are to being over-medicated, with the complications…
It’s been nearly a year since the EveryLife Foundation for Rare Diseases released its expansive report finding the total economic burden of rare disorders in the U.S. to be nearly $1 trillion.
Folate produced by a specific kind of gut bacteria likely contributed to weight loss in a teenager with Prader-Willi syndrome (PWS) who was given a high-fiber diet for several months. A trio of researchers at Shanghai Jiao Tong University in China described the case in a study, “…
Several characteristics during pregnancy and immediately after birth — such as maternal age, pregnancy complications, low birth size, reduced muscle tone, and infant feeding difficulties — may distinguish a newborn with Prader-Willi Syndrome (PWS) from healthy babies early in life, a study showed. “By providing an overview of…
Since 2008, Rare Disease Day — the last day of February — has brought together patients, caregivers, family members, friends, and advocates from around the world to raise awareness and improve equity for the more than 7,000 known rare diseases that affect more than 300 million people. In 2022, the…
Researchers have documented the first case of a rare condition characterized by neurocognitive regression and epileptic seizures during sleep in a child with Prader-Willi syndrome (PWS). Their case report, “Electrical status epilepticus during sleep in a child with Prader-Willi syndrome: a case report,” was published in…
A Phase 2 study evaluating ARD-101, an investigational, gut-targeting Prader-Willi syndrome (PWS) oral therapy, is now enrolling up to 12 patients, ages 17 to 65. The open-label study, sponsored by Aardvark Therapeutics, the treatment’s developer, will be held at two sites: Children’s Hospital in Colorado, which is…
Researchers have developed guidelines for screening and treating hypogonadism, or underdeveloped sex organs, in women with Prader-Willi syndrome (PWS), noting this condition is frequent in these patients, but often left undiagnosed. Given that the resulting low levels of sex hormones can cause osteoporosis (weak…
Levels of several hunger-regulating hormones are dysregulated in people with Prader-Willi syndrome (PWS), which likely contributes to the excessive hunger that characterizes the condition, a study suggests. Clustering patients based on these hormone levels could help to detect PWS and identify targets for therapies, researchers say. The study, “…
Radius Health is sharing more details about RAD011, its investigational cannabidiol-based oral therapy meant to reduce excessive appetite, or hyperphagia, in people with Prader-Willi syndrome (PWS). Its fact sheet contains not only results from previous preclinical and clinical studies, but also the design of the upcoming…
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