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A six-week remote parent-training intervention for children with Prader-Willi syndrome (PWS) got good acceptability and satisfaction scores from parents, supporting the use of telehealth to manage the social and emotional impairments of children with this rare condition, a study reports. The study, “The PRETEND Program: Evaluating the Feasibility of…
Impairments in behavioral and working memory tasks are seen in people with Prader-Willi syndrome (PWS) compared with healthy controls, a new study shows. The study, “Investigation of the relationship between electrodermal and behavioural responses to executive tasks in Prader-Willi syndrome: An event-related experiment,” was published in the journal…
Liza Zickefoose has six children. As if that’s not challenging enough, one of them, Bridgette Nicole Johnston, has Prader-Willi syndrome (PWS). But from Zickefoose’s perspective, her daughter has a special purpose — to teach people of God’s love. That epiphany led to her writing a picture book titled “More…
Neonatal hypotonia (low muscle tone), reduced fetal movement, feeding difficulties, and cryptorchidism (undescended testes) should immediately suggest a diagnosis of Prader-Willi syndrome (PWS), according to a recent study. “Neonatal Features of the Prader-Willi Syndrome; The Case for Making the Diagnosis During the First Week of Life,” was published in…
Saniona announced it has completed the second part of its exploratory Phase 2a study testing Tesomet (tesofensine/metoprolol) as a therapy for Prader-Willi syndrome, involving nine adolescent patients treated for three months. The treatment seemed to be well-tolerated, and eight of the nine participants agreed to continue in a 24-week open…
A Phase 3 trial of Levo Therapeutics‘ treatment candidate LV-101 (intranasal carbetocin) is enrolling participants ages 7-18 with Prader-Willi syndrome. Patients are being recruited at the University of Florida and Vanderbilt University, with Levo anticipating additional study sites to open. Updates on trial sites can be found…
Cash-strapped governments across the 28-member European Union are struggling to control runaway healthcare expenditures — at exactly the same time as the promise of new but expensive therapies to treat rare diseases has never been greater. That’s the paradox faced by pharmaceutical companies as well as patient advocacy groups in…
The U.S. Food and Drug Administration has granted an orphan drug designation to Auris Medical for its intranasal betahistine product for the treatment of obesity associated with Prader-Willi syndrome. In addition to Prader-Willi syndrome, the company also has plans to expand the betahistine development program to include depression…
Aripiprazole, a medication used to treat the symptoms of schizophrenia and other psychotic disorders, is a potential therapeutic option for patients with Prader-Willi syndrome and psychosis, a new case report suggests. The study, “Clinical Usefulness of Aripiprazole Treatment in a Girl…
Treatment with Soleno Therapeutics’ diazoxide choline controlled release (DCCR) tablets reduced excessive appetite and lowered body fat in Prader-Willi syndrome (PWS) patients in a Phase 2 trial, the company announced. The latest findings were presented during the Obesity Society Meeting 2018 in Nashville, Tennessee, in the poster, “…
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