Author Archives: Marta Figueiredo PhD

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…

Tonix Pharmaceuticals has entered a research agreement with Inserm Transfert, the private subsidiary of the French National Institute of Health and Medical Research (Inserm), to study the mechanisms behind the reported benefits of the oxytocin hormone in a mouse model of Prader-Willi syndrome (PWS). Inserm Transfert signed the deal…

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…

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…

Pediatric multidisciplinary team clinics for Prader-Willi syndrome (PWS) offer an improved service that supports the diverse and challenging needs of children with the disease, according to a small parental interview-based U.K. study. Parents reported that this type of clinic enabled them to address issues other than health, such as…

The activity of nine genes located outside the so-called PWS locus — a region containing genes in Prader-Willi syndrome (PWS) that are lost or have defects — is significantly altered in people with the disease, according to a study in lab-grown nerve cells derived from patients and healthy individuals.

Infants and toddlers with Prader-Willi syndrome (PWS) have significantly lower blood levels of insulin-like growth factor 1 or IGF-1 — indicative of growth hormone (GH) deficiency — than their healthy peers, a small study in Japan shows. This is the first study to conduct this type of comparative analysis…

A multidisciplinary approach that included weight reduction through calorie restriction and diuretics, ventilation support, skin care, and rehabilitation successfully managed life-threatening right heart failure due to severe obesity in a young man with Prader-Willi syndrome (PWS). These findings from a case study highlight not only the effectiveness of such a…

There is no substantial evidence supporting the efficacy of LV-101 (intranasal carbetocin), a nasal spray designed to reduce the insatiable hunger, called hyperphagia, that’s a hallmark of Prader-Willi syndrome (PWS), according to a 12-1 vote by a U.S. Food and Drug Administration (FDA) advisory committee. The overwhelmingly negative vote followed…