Category: Research

Saniona Initiates Phase 2b Clinical Trial of Tesomet for Prader-Willi Syndrome

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...

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...

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...

Scroll to top