The first healthy volunteers were dosed in a Phase 1 clinical trial evaluating Inversago Pharma‘s INV-102, a potential therapy for Prader-Willi syndrome (PWS) and other metabolic disorders. According to a company press release, the trial, underway in Canada, started dosing following a “No Objection Letter” from Health Canada.
News
The U.S. Food and Drug Administration (FDA) is holding a public advisory committee meeting regarding LV-101 (intranasal carbetocin) as a potential treatment of distress behaviors and insatiable hunger in Prader-Willi syndrome (PWS) patients. The Psychopharmacologic Drugs Advisory Committee meeting, to be held virtually Nov. 4 from 10…
One year of treatment with Soleno Therapeutics’ investigational oral therapy diazoxide choline controlled release, known as DCCR, significantly reduced excessive appetite and disease-related behaviors in Prader-Willi syndrome (PWS) patients, according to top-line data from an extension study of the DESTINY PWS Phase 3 trial. These trajectories were significantly…
People with Prader-Willi syndrome (PWS) have smaller pituitary glands than their typically-developing peers, according to data from a small study. The findings also suggest that altered pituitary size is associated with behavioral abnormalities in PWS. Results were published in the journal Biomarkers in Neuropsychiatry, in the study “…
The mean total annual costs of raising a child with Prader-Willi Syndrome (PWS) — an estimate that takes into account services use, impact on parental income, and care arrangements — is more than AU$57,500 (about $42,500) per child, according to an Australian study of four rare disorders that cause…
An $11.2-million, five-year grant has been awarded by the Centers of Excellence in Genomic Science of the National Institutes of Health (NIH) to uncover high-resolution details of the entire human genome. This project should help better understand the genome’s structure and function, as well as provide important clues about the…
Vision problems such as lazy eye, farsightedness, and strabismus — when the eyes don’t line up in the same direction — are more common in people with Prader-Willi syndrome (PWS) than among their healthy peers, a study shows. These findings, along with data showing that vision problems may negatively affect…
Children and adults with Prader-Willi syndrome (PWS) are not at higher risk of developing a severe COVID-19 infection, according to results from a survey involving more than 500 patients in France. These findings were particularly surprising in terms of the adult population, which typically has several known risk factors for severe…
Pitolisant, a treatment for narcolepsy, should be considered for people with Prader-Willi syndrome (PWS) whose response to psychotropic medications is less than optimal, a case report of a 15-year-old patient suggests. “Pitolisant has the potential to improve many symptoms in patients with Prader-Willi syndrome and it appears to be…
The hunger hormone ghrelin, when measured in infancy, may be a biomarker for predicting the early onset of scoliosis — a sideways curvature of the spine that looks like the letter C or S — in children with Prader-Willi syndrome (PWS), a study suggested. Beginning in early infancy,…
Recent Posts
- Setting boundaries at home and work helps me as a PWS caregiver
- FDA lifts hold on clinical testing of experimental PWS treatment
- Redefining the menu to keep the magic of Halloween alive with PWS
- PWS doctors skip sleep studies for early growth hormone therapy
- We all ‘come out’ in our own ways, including PWS families
- Testosterone therapy aids bone health in PWS, with low behavior impact
- Why common illnesses can look different with Prader-Willi syndrome
- Getting away is hard for couples raising a medically complex child
- Interdisciplinary inpatient care may boost outcomes in PWS patients
- Staying present is a challenge when you know what PWS might bring next