Category: Research

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...

September Is Newborn Screening Awareness Month

What is newborn screening? Newborn screening is one of the most successful public health programs ever enacted, saving thousands of lives over the past 50 years. Newborn screening allows physicians to catch rare genetic conditions at birth and start treatment almost immediately following birth; it enables efficient diagnosis and access to treatments that can save...

Prader-Willi Syndrome Association | USA and the Foundation for Prader-Willi Research Partner for Telehealth Needs Assessment

Prader-Willi Syndrome Association | USA (PWSA | USA) and the Foundation for Prader-Willi Research (FPWR) have partnered to conduct a study to measure interest in telehealth as a way of increasing access to care from Prader-Willi syndrome specialists. In addition, the project aims to better understand the demographic makeup of the Prader-Willi syndrome (PWS) parent...

Prader-Willi Syndrome and Gastroparesis

There is little evidence in the literature regarding the incidence of gastroparesis in PWS and how it affects families in the PWS community. Within the Global PWS Registry, 13% of participants have been diagnosed with gastroparesis (N=739). Of those with gastroparesis, more than half were diagnosed under age 10. The severity of gastroparesis reported ranges...

Levo Therapeutics Announces Top-line Results from Phase 3 CARE-PWS Study of LV-101 (Intranasal Carbetocin) for the Treatment of Prader-Willi Syndrome

CHICAGO–(BUSINESS WIRE)–Levo Therapeutics, Inc., a biotechnology company dedicated to using genetic insights to advance treatments for Prader-Willi syndrome (PWS) and related disorders, announced today top-line results from the Phase 3 CARE-PWS clinical study evaluating LV-101 (intranasal carbetocin) for the treatment of PWS. This syndrome is a complex, neurodevelopmental disorder that occurs in approximately 1 in...

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