Contributed by Stacy Ward, MS Director of Family Support and Lynn Garrick, RN, BSN Medical/Research Coordinator Prader-Willi syndrome (PWS) is a rare neurodevelopmental genetic disorder that affects multiple systems in the body. There are many symptoms of PWS, including hyperphagia, behavioral challenges, hypotonia, incomplete sexual development, cognitive deficits, metabolic dysregulation, and several more. Hyperphagia is...
Category: Medical
Clinical Trial Sites Announced for Radius Health’s RAD011 SCOUT-015 Study
Radius Health has announced the locations for its RAD011 SCOUT-015 Clinical Trials. Below, you will find a downloadable map with each participating hospital and medical center around the country. The RAD011 SCOUT-015 studies are in various stages, depending on the location, from initiated to activated and ready to enroll. You can also find a comprehensive...
World Osteoporosis Day
Read PWSA | USA Clinical Advisory Board's full Consensus Statement Here. Wednesday, October 20th is recognized as World Osteoporosis Day. Osteoporosis is a condition that commonly impacts individuals living with Prader-Willi syndrome, and is typically diagnosed in adolescence and adulthood. The cause(s) of the osteoporosis is not totally clear, but it is thought to be...
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...
Soleno Therapeutics Announces Positive Data Showing Continued Significant Improvements in Symptoms of PWS following One Year Treatment with DCCR
(Soleno Therapeutics Press Release) Statistically significant reduction in hyperphagia and all other PWS behavioral parameters in Study C602 Statistically significant improvements compared to natural history of PWS from the PATH for PWS Study On track for data submission to the FDA in Q3 2021 REDWOOD CITY, Calif., Sept. 08, 2021 (GLOBE NEWSWIRE) -- Soleno Therapeutics,...
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,...
FDA Grants Priority Review for Levo Therapeutics’ New Drug Application for LV-101 (Intranasal Carbetocin) for the Treatment of Prader-Willi Syndrome
Read Levo Therapeutic, Inc.’s Full Article HERE. CHICAGO, IL, July 6, 2021 (Newswire.com) – Levo Therapeutics, Inc., a biotechnology company dedicated to using genetic insights to advance treatments for Prader-Willi syndrome (PWS) and related disorders, announced today that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for review and...
Sleep Disturbances in Prader-Willi Syndrome
Sleep health is essential for everyone; it is just as important to take care of ourselves as parents and caregivers as it is for those living with Prader-Willi syndrome (PWS). We understand that disordered sleep has implications for cognitive outcomes, mental and physical health, and work and school performance. Sleep disturbances can occur from many...
News Release: Rhythm Wins FDA Approval for Obesity Drug Imcivree
Rhythm Pharmaceuticals Inc., a company developing medicines for rare genetic disorders of obesity, has won FDA approval for its first product, Imcivree (setmelanotide), following a priority review. The drug is designed to restore a biological pathway that, when disrupted, can lead to constant hunger. The approval covers the treatment of three types of ultrarare early...
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...