News

Starting treatment with growth hormone early in life — before age 2 — was associated with better cognitive and behavioral outcomes for children with Prader-Willi syndrome (PWS) in a recent study out of Spain. “We found greater cognitive and adaptive performance in patients who had received early treatment,” its…

The National Organization for Rare Disorders (NORD) has updated its State Report Card to make it more digitally friendly and added telehealth to its categories of rare disease policy issues in a nod to its increased use during the ongoing COVID-19 pandemic. NORD’s report card project began seven…

Saniona will pause the Phase 2b clinical trials testing Tesomet for people with Prader-Willi syndrome (PWS) and hypothalamic obesity (HO). The decision “is not related to the safety or efficacy of Tesomet and is entirely due to funding limitations,” the company stated in a press release. The…

Impaired social responsiveness, an indicator of autism spectrum disorder (ASD), is prevalent among adults with Prader-Willi syndrome (PWS), and is associated with worse social functioning, according to a recent study. “Our results suggest that evaluating difficulties in social skills is fundamental for the global assessment of functionality,” the researchers…

People with Prader-Willi syndrome (PWS) who had COVID-19 experienced either no symptoms or only mild illness due to the infection, an international survey study found. These findings were unexpected, as researchers had put forward the hypothesis that the presence of other conditions typically coexisting with PWS would make symptoms…

Children, adolescents, and adults with Prader-Willi syndrome (PWS) have distinct craniofacial patterns relative to healthy individuals, according to a study in Norway. These patterns also changed with age, with children showing a more receding chin, and adults exhibiting a prognathic, or forward-projecting lower jaw, along with abnormal tooth angles. Findings from…

Patient registries are a hot topic of rare disease research and many organizations are taking advantage of this resource by signing up their patient communities and connecting with researchers. Eric Sid, MD, program officer for the Office of Rare Diseases Research (ORDR), said it is difficult to estimate how…

Soleno Therapeutics has announced that its diazoxide choline controlled release (DCCR) tablets will be evaluated in a clinical trial for people with early-phase Prader-Willi Syndrome (PWS), which will be funded by Running for Research – Prader-Willi Syndrome, an ongoing fundraiser to benefit PWS research operated by the University…

Recent changes to a Phase 2 clinical trial of the oral medicine pitolisant to treat excessive daytime sleepiness will make it easier for people with Prader-Willi syndrome (PWS) to participate in the study, according to an announcement from the Foundation for Prader-Willi Research. “In addition to evaluating…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to TNX-2900, Tonix Pharmaceuticals’ oxytocin therapy for Prader-Willi syndrome (PWS). Orphan designation is intended to speed the development of potential therapies for rare diseases, or those affecting fewer than 200,000 people in the U.S. The status…