Category: Research

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...

Statement from Members of the Clinical Advisory Board (CAB) Regarding People with PWS and COVID-19 Immunization  

The types of vaccines now available against COVID-19 use mRNA technology.  This type of vaccine has been under development for many years and, unlike other immunizations, does not place a weak or inactive germ into the body, but instead teaches cells in our body to make an immune response that then produces antibodies which provide protection if...

Harmony Biosciences Enrolls First Patient in Phase 2 Trial Evaluating Pitolisant For Excessive Daytime Sleepiness in Patients with Prader-Willi Syndrome

PLYMOUTH MEETING, PA and CHICAGO, IL, December 15, 2020 — Harmony Biosciences Holdings, Inc. (“Harmony”) (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients living with rare neurological disorders who have unmet medical needs, today announced the first patient has been enrolled in a Phase 2 trial evaluating the safety...

Scroll to top