Results from a Phase 2 clinical trial show that Levo Therapeutics’ lead therapy candidate, LV-101 (intranasal carbetocin), can effectively reduce symptoms of Prader-Willi syndrome (PWS) in children, the company announced. The investigational therapy was found to decrease the patients’ uncontrolled urge for food and excessive eating, with a…
News
Patients with Prader-Willi Syndrome (PWS) have lower levels of the angiopoietin-like 8 protein (ANGPTL8), which may be a marker for liver disease, than individuals of similar age and size who don’t have PWS, research shows. The results were reported in the study “Circulating angiopoietin-like 8 (ANGPTL8) is…
A new study shows that alterations in body composition, appetite-regulating factors, cardiovascular disease markers, and bone density are common in adults with Prader-Willi syndrome (PWS). The study, titled “Body composition, adipokines, bone mineral density and bone remodeling markers in relation to IGF-1 levels…
A new video explains how Prader-Willi syndrome (PWS) develops, some of its challenges, and ongoing research — all in just under three minutes. Titled “What is Prader-Willi Syndrome?” the video is narrated by Susan Hedstrom, executive director of the Foundation for Prader-Willi Research (FPWR) and the mother of a…
Saniona‘s investigative treatment for Prader-Willi syndrome, Tesomet tablets, were well-tolerated in preclinical toxicology studies, the company announced. The positive results support the safe dosing of Tesomet in long-term Phase 2 and Phase 3 clinical trials, the company said. Results also revealed that Tesomet, a fixed-dose combination of tesofensine…
As President Trump signed the recently passed Right to Try legislation into law in a White House ceremony, Jordan McLinn of Indianapolis tried twice to embrace him. The third time, 9-year-old Jordan finally got the hug he wanted — as well as a kiss on the forehead. The video of…
Biotech company Saniona recently announced that the Phase 1 pharmacokinetic study of a new tablet therapy candidate for Prader-Willi syndrome, called Tesomet (tesofensine/metoprolol), produced promising results in healthy men. Prader-Willi syndrome (PWS) is an inherited disorder that creates feelings of constant hunger, which often leads to obesity and type 2 diabetes.
Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a rather nebulous term that means different things to different people. “Recently, there’s been much more of a focus on Quality of Life (QoL) issues, real-world evidence and patient-reported outcomes,” said…
Perinatal complications, including hypotonia (low muscle tone) and feeding difficulties, are clinical features more common among newborns with Prader-Willi syndrome, compared with the general population, according to a new study. The study with that finding, “Multicentre study of maternal and neonatal outcomes in individuals with Prader-Willi syndrome,”…
Motor disability is a common problem in patients with Prader-Willi syndrome (PWS) and may negatively impact their caregivers’ quality of life, particularly physical and emotional problems, researchers from Taiwan reported. Their study, “Motor performance in Prader-Willi syndrome patients and its potential influence on caregiver’s…
Recent Posts
- Still-recruiting ARD-101 trial for PWS lowers US enrollment age to 7
- For rare disease families, February is a month of both love and awareness
- Muscle, bone signaling imbalances found in non-obese PWS children
- Most PWS patients meet nutrient targets but struggle with calorie control
- Adults with PWS need lifelong support in daily activities, study finds
- Celebrating a milestone in life with Prader-Willi syndrome
- New technique ‘wakes up’ silent genes in Prader-Willi in lab testing
- Probiotics may boost beneficial gut bacteria in Prader-Willi syndrome
- Avoiding meltdowns during the holidays with Prader-Willi syndrome
- PWS patients see weight loss, less hunger with setmelanotide in trial