News

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…

The U.S. Food and Drug Administration (FDA) continues to call for an additional clinical trial of Soleno Therapeutics’ diazoxide choline controlled release (DCCR) tablets before considering it for approval for Prader-Willi syndrome (PWS). However, the agency agreed that such a trial, if appropriately designed, may involve patients currently receiving the…

The U.S. Food and Drug Administration (FDA) has told Levo Therapeutics that an additional clinical trial will be necessary to confirm the efficacy of LV-101 (intranasal carbetocin), a nasal spray aimed at reducing insatiable hunger, called hyperphagia, in Prader-Willi syndrome (PWS) patients. Levo Therapeutics had submitted a new drug…

A low-cost screening test called methylation-specific quantitative melt analysis (MS-QMA) could feasibly be used to conduct newborn screening for Prader-Willi syndrome (PWS) and related conditions, according to a new study. The study, “Feasibility of Screening for Chromosome 15 Imprinting Disorders in 16579 Newborns by Using a…