Via Saniona: PRESS RELEASE December 28, 2021 Saniona (OMX: SANION), a clinical-stage biopharmaceutical company focused on rare diseases, today announced the initiation of a Phase 2b clinical trial of Tesomet in patients with Prader-Willi syndrome (PWS). Tesomet is an investigational fixed-dose combination therapy of tesofensine, a triple monoamine reuptake inhibitor, and metoprolol, a beta-1 selective blocker. Data from the...
Category: Research
The Chicago School of Professional Psychology is looking for Research Study Participants
YOU can help the Chicago School of Professional Psychology learn about the effects of a behavioral caregiver training program for caregivers of children with PWS. Participants will be compensated with a $500 Visa gift card following the completion of the study. Parents with children with multiple diagnoses can reach out to Dr. Bedard for clarification...
Research Survey Opportunity: Behavioral Supports and Services for Children with Prader-Willi Syndrome
Are you a caregiver of at least one child, of any age, who has been diagnosed with Prader-Willi Syndrome? Do you have access to the internet and an internet-connected device? Can you read and write in English? Are you over the age of 18? If you answered YES to these questions, you are invited to...
Prolapsed Rectum and Risk Factors in Prader–Willi Syndrome: A Case-Based Review
Written by: Merlin G. Butler ABSTRACT A 14-year-old boy with Prader–Willi syndrome (PWS) with maternal disomy 15 is reported with rectal prolapse as only the second patient in the literature. With predisposing risk factors present for rectal damage and prolapse in this syndrome, the incidence must be higher and therefore underreported. These risk factors include...
Research Opportunity: Project Pathways
The Learning Lab for Intellectual and Developmental Disabilities at the University of Nebraska – Lincoln has reached out to us asking for any families interested in participating in a study they’re currently working on titled, Project Pathways. The purpose of this study is to assess the reading and writing profiles of students with intellectual and...
Summary of a Streamlined Molecular Diagnostic Approach for Prader-Willi and Other Related Syndromes
Written by: Merlin G. Butler, MD, PhD Historically, to confirm the diagnosis and molecular genetic classes in Prader Willi syndrome (PWS) required a stepwise approach using multiple methods needing more time and resources. Due to advances in genetic testing and availability of multiple analytical methodologies, a streamlined approach was developed and reported by Strom et...
FDA Advisory Committee to Review LEVO’s Carbetocin as a Treatment for PWS
We are excited to share that Levo’s New Drug Application (NDA) for carbetocin as a treatment for PWS will be discussed at a public meeting of the Psychopharmacologic Drugs Advisory Committee to be held November 4, 2021. This is a major step forward on this drug's path through the approval process. The Food and Drug...
Harmony Biosciences Acquires Asset with Novel Mechanism of Action for the Potential Treatment of Narcolepsy and other Rare Neurological Diseases
Harmony Biosciences recently announced the acquisition of HBS-102, a potential first-in-class molecule with a novel mechanism of action, from ConSynance Therapeutics, Inc. HBS-102 is a Melanin Concentrating Hormone Receptor 1 (MCHR1) antagonist that has the potential to offer a novel approach to the treatment of narcolepsy including the symptoms of Rapid Eye Movement (REM) sleep...
Harmony Biosciences adds Cincinnati, OH site to its Phase 2 Clinical Trial
Harmony Biosciences is currently in its Phase 2 clinical trial of Pitolisant, studying the safety and impact of an investigational medicine for excessive daytime sleepiness, cognition, and behavioral function in people with PWS. This week, they added an 11th location to their list of trial sites around the U.S. The trial site will be located...
Radius Health Announces Plans for Global Prader-Willi Syndrome Pivotal Study
On behalf of Radius Health, Inc: Boston, Mass., July 22, 2021 — Radius Health, Inc. announced Wednesday, July 21, 2021 that it has recently received the written meeting minutes from a June Type C meeting held with the U.S. Food and Drug Administration (FDA) regarding RAD011, a synthetic cannabidiol oral solution. RAD011 is initially to...
Soleno Therapeutics Provides Update on DCCR for the Treatment of PWS
FDA agrees to review additional data to determine adequacy for submission of NDA Soleno Therapeutics, Inc. provided an update on Tuesday, July 7, 2021 following a recent interaction with the FDA regarding the development of DCCR, a potential treatment for PWS. According to Soleno Therapeutics, on July 2, 2021 they received news from The FDA,...
Harmony Biosciences is seeking people with PWS between the ages of 6 – 65 to enroll in Clinical Trial
Click Here to View and Download Harmony Biosciences’ PWS Trial Infographic