News

The U.S. Food and Drug Administration has placed a full clinical hold on late-stage clinical trials of Aardvark Therapeutics’ oral therapy candidate ARD-101 in patients with Prader-Willi syndrome (PWS) and hyperphagia, or insatiable hunger. The company had voluntarily paused…

Tonix Pharmaceuticals has pushed back the planned start of a Phase 2 clinical trial testing its oxytocin-containing TNX-2900 nasal spray in children and adolescents with Prader-Willi syndrome (PWS). Tonix had expected the U.S.-based study, which has been cleared by the U.S. Food and Drug Administration (FDA), to start…

Infants younger than 6 months with Prader-Willi syndrome (PWS) showed increased blood flow in brain regions involved in movement, motivation and reward, emotional processing, and social behaviors, a study in France reports. Data also showed that blood flow in one brain region was associated with oral-motor performance, including sucking…

May is Prader-Willi Syndrome (PWS) awareness month, offering families and advocates an opportunity to raise visibility, share experiences, and drive change for those affected by the disease. Through the United in Action initiative, launched by the PWS Association USA (PWSA USA), supporters are encouraged to turn everyday connections…

Researchers have developed a one-time gene therapy that may help ease obesity and related metabolic problems in people with Prader-Willi syndrome (PWS), according to a study in a mouse model of the disease. A single injection of the therapy, which uses a harmless version of the adeno-associated virus (AAV)…

A new study uses a mobile app to track the experiences of caregivers for people with Prader-Willi syndrome (PWS), with the aim of giving researchers insight into day-to-day life that could inform treatment development. “Pragmatic measurement is an important key to drug development that can provide insight into the…

More than half of children with Prader-Willi syndrome (PWS) show signs of spontaneous puberty, but more than three-quarters will have hypogonadism (reduced function of the reproductive organs), according to a study in Australia. That means patients generally will not attain full sexual maturity without therapies to normalize hormone levels.

Typically developing siblings of children with Prader-Willi syndrome (PWS) often report feeling misunderstood and isolated, a new review highlights. The results underscore that PWS care needs to support not only the child with the condition and their parent(s), but also the entire family system. “Family context shapes sibling experiences…

Aardvark Therapeutics has provided new details on the safety concerns that led to the pause of a global clinical trial of ARD-101, its experimental treatment for hyperphagia, or persistent, insatiable hunger, in people with Prader-Willi syndrome (PWS). In a company press release announcing financial results and the…

Telemental health programs, which provide remote mental health services, may be a feasible and effective strategy to address mental health issues that arise for caregivers of children with rare diseases, including Prader-Willi syndrome (PWS), a U.S. study shows. In particular, the researchers noted that individual and group-based programs were…