Soleno Therapeutics announced positive top-line results from their randomized Study C602 withdrawal period assessing the use of diazoxide choline (DCCR) extended-release tablets for the treatment of Prader-Willi syndrome (PWS). The results, announced via news release, support Soleno’s planned submission of a New Drug Application to the US Food and Drug Administration (FDA) in 2024. PWS…
The Kaufman Brief Intelligence Test may be a reliable alternative to conventional tools for evaluating intellectual cognition in people with Prader–Willi syndrome (PWS), according to a study published in the Journal of Intellectual Disability Research. The researchers recruited patients with a genetically confirmed diagnosis of PWS who regularly received transdisciplinary treatment at an institution for…
Primary caregivers of patients with Prader-Willi syndrome (PWS) often experience significant deterioration in their mental health and quality of life, according to a study published in Child Care, Health and Development. PWS is a rare disorder linked to developmental delay, intellectual disability, and behavioral issues, including at times self-injurious behaviors. Hyperphagia, a prominent feature of…
Employment hours and family environment factors might be significant predictors of compliance in a 24-week family home-based physical activity intervention in families with children who have Prader-Willi syndrome (PWS), according to a study published in Obesity Pillars. The goal of this study was to determine which parental and familial factors predicted completion of and compliance…
Researchers presented the case report of a boy with Prader-Willi syndrome (PWS) who developed vagal asystole in Pediatrics. PWS is a hormonal disorder of the hypothalamic-pituitary-axis caused by microdeletions within chromosome 15. Despite advancements in care, patients with PWS still have greater morbidity and mortality compared with the general population. Although severe cardiological malformations are…
The US Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation for oral denatonium acetate (ARD-101) to treat Prader-Willi syndrome (PWS), Aardvark Therapeutics announced. The designation, announced via news release, is accompanied by a phase 2 trial of oral ARD-101 that is now open for additional enrollment of participants. PWS is a severe…
Harmony Biosciences announced the successful completion of an end-of-phase 2 meeting with the US Food and Drug Administration (FDA) regarding pitolisant as a therapy for excessive daytime sleepiness (EDS) in Prader-Willi syndrome (PWS). The announcement, via news release, includes plans to launch a phase 3 registrational study in late 2023. “Building upon the encouraging data…
In Prader-Willi syndrome (PWS), neuronal excitabilities and mutant neurons demonstrate substantially lower intensity than that of control neurons, according to a study published in Scientific Reports. PWS is a genetic disorder that results in significant obsessive-compulsive and psychosis-related features in adult patients. These psychiatric manifestations have been linked to autism spectrum disorder (ASD). “Given that…
A new study has identified contributing factors, including sensorimotor processes, associated with impaired postural stability in adults with Prader-Willi syndrome (PWS). The study, published in Human Movement Science, found that postural impairments in these patients were generally not linked to obesity status. “This study comprehensively evaluated physical function in adults with PWS by determining the…
Patients with Prader-Willi syndrome commonly experience serious medical events, according to a presentation at a PWS association’s national convention.
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