News

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…

The U.S. Food and Drug Administration (FDA) has told Levo Therapeutics that an additional clinical trial will be necessary to confirm the efficacy of LV-101 (intranasal carbetocin), a nasal spray aimed at reducing insatiable hunger, called hyperphagia, in Prader-Willi syndrome (PWS) patients. Levo Therapeutics had submitted a new drug…

A low-cost screening test called methylation-specific quantitative melt analysis (MS-QMA) could feasibly be used to conduct newborn screening for Prader-Willi syndrome (PWS) and related conditions, according to a new study. The study, “Feasibility of Screening for Chromosome 15 Imprinting Disorders in 16579 Newborns by Using a…

In people with Prader-Willi syndrome (PWS), certain parts of the brain associated with decision-making and reward processing are less active in response to the sight of food, a study has found. The findings suggest that people with PWS get less of a “reward” feeling from eating than their typically…

Adolescents and adults with Prader-Willi syndrome (PWS) say they struggle with constant hunger and the urge to seek food, according to an interview-based study. Participants experienced difficulties with anxiety, behavioral outbursts, changes in schedule, and schooling, with many trying to distance themselves from the syndrome. They also expressed 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…

Patients with Prader-Willi syndrome (PWS) are at increased risk of developing type 2 diabetes mellitus as they transition into adulthood, particularly if they are severely obese and/or undergoing rapid weight gain, according to a recent study. “These results underline the need for early identification of patients at risk, prevention…

Saniona is launching a Phase 2b clinical trial of Tesomet to evaluate its potential in lessening the uncontrollable hunger of people with Prader-Willi syndrome (PWS). “The initiation of this Phase 2b clinical trial of Tesomet in Prader-Willi syndrome follows our recent initiation of a Phase 2b clinical…

The National Organization for Rare Disorders (NORD) asks Americans to plan ahead to participate in the Light Up for Rare campaign to raise awareness of rare diseases. NORD is the U.S. sponsor for Rare Disease Day on Feb. 28. The annual awareness day spotlights approximately 7,000…

Two boys with Prader-Willi syndrome (PWS) developed respiratory failure — a condition in which the oxygen level in the blood becomes too low — after surgery to correct scoliosis (a sideways curvature of the spine), a report says. “Even after scoliosis surgery, ‘periodic’ monitoring of respiratory failure with an…