News

Bright Minds Biosciences has launched a clinical program to evaluate whether its oral therapy candidate BMB-105 can ease hyperphagia, or insatiable hunger, and other behavioral symptoms in people with Prader-Willi syndrome (PWS). The experimental therapy targets the underlying disease mechanisms of PWS, and as such, is a “novel”…

Long-term treatment with human growth hormone, also known as somatropin, does not seem to influence mortality in people with Prader-Willi syndrome (PWS), but may increase the risk of developing type 2 diabetes, a study from South Korea found. The risk of type 2 diabetes increased by 6%…

Prader-Willi syndrome (PWS) is associated with shorter life expectancy, impaired quality of life, and high healthcare and caregiving costs — all of which place a heavy burden on families and the healthcare system. That’s according to a systematic review of more than 130 published studies. Data also found that…

Once-daily treatment with pitolisant is generally safe and helps reduce excessive daytime sleepiness (EDS) and abnormal behaviors in people with Prader-Willi syndrome (PWS) — notably with the repurposed therapy given at higher doses, and especially in children. That’s according to the full results of a proof-of-concept trial that tested two…

Soleno Therapeutics plans to help accelerate research toward finding a genetic cure for Prader-Willi syndrome (PWS). “We will commit up to $5 million in grants in the next year, where we will find the most promising genetic approaches to treat PWS, and find the ones where further significant investment…

Aardvark Therapeutics has amended the protocol for the Phase 3 HERO clinical trial testing ARD-101, its investigational oral therapy for excessive hunger associated with Prader-Willi Syndrome (PWS), to include patients as young as 10 years old. “Expansion of the Phase 3 HERO trial to include children 10 years…

Tonix Pharmaceuticals is planning the launch of a Phase 2 clinical trial to test its TNX-2900 nasal spray — containing a specialized form of oxytocin — in children and teens with Prader-Willi syndrome (PWS). The company has already received clearance from the U.S. Food and Drug Administration (FDA) to…

A Phase 3 clinical trial testing ACP-101 for hyperphagia, or uncontrollable hunger, in Prader-Willi syndrome (PWS) patients failed to meet its primary and secondary goals. Top-line results from the trial of Acadia Pharmaceuticals’ experimental nasal spray, dubbed COMPASS PWS (NCT06173531), showed no significant differences between the ACP-101…

Targeting an enzyme called EHMT2 may help treat Prader-Willi syndrome (PWS) by reactivating the maternal PWS genes that are normally turned off, according to a new study. PWS is caused by the loss of certain genes in a region of the paternal chromosome 15 called the PWS locus.

Adults with Prader-Willi syndrome (PWS) caused by a genetic deletion have higher obesity rates and more severe obesity than those with PWS associated with other genetic abnormalities, a study found. “Given the higher risk of obesity observed in individuals with the deletion subtype, our results support the need for…