News

December 17, 2020December 17, 2020

Soleno Shares FDA’s Feedback on Potential PWS Therapy

News

Soleno Therapeutics has received the official feedback from its meeting with the U.S. Food and Drug Administration (FDA) regarding next steps for diazoxide choline controlled-release (DCCR) tablets as a ... Read more

December 10, 2020December 10, 2020

Early, Sustained Growth Hormone Treatment Linked to Greater Benefits in Real-world Study

News

Growth hormone (GH) therapy initiated at an earlier age and sustained for a longer period results in greater improvements in growth and body composition in children with Prader-Willi syndrome (PWS), ... Read more

December 8, 2020December 8, 2020

Mediterranean Diet, Exercise Reduce Weight, Cholesterol Levels in PWS Patients, Study Shows

News

A three-week program based on Mediterranean diet and exercise, and performed three times in six years, led to significant weight loss and reduced cholesterol levels in adults with Prader-Willi ... Read more

December 4, 2020December 4, 2020

NORD Push for Rare Disease Advisory Councils Focus of Dec. 16 Public Meeting

News, syndicated

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders ... Read more

December 3, 2020December 3, 2020

Genetic Subtypes Determine Frequency of Psychiatric Behaviors in PWS, Study Finds

News

The frequency of skin picking and anxiety in people with Prader-Willi syndrome (PWS) depends on their specific type of genetic defects, an eight-year study found. Treatment with growth hormone (GH) ... Read more

December 1, 2020December 1, 2020

PWSA USA Hosting Dec. 13 Virtual ‘Hope for the Holidays’ Benefit Concert

News

The Prader-Willi Syndrome Association (PWSA) USA, a nonprofit organization that supports education, advocacy, and awareness for those with the complex genetic disorder, announced that it will hold its first ... Read more

November 26, 2020November 26, 2020

Survey Finds COVID-19 Disrupted Care, Well-Being of Rare Disease Patients in Europe

News, syndicated

The first wave of COVID-19 in Europe severely disrupted access to care and raised stress and anxiety in people with rare diseases, negatively affecting their health and well-being, according to ... Read more

November 24, 2020November 24, 2020

Gene Silencing Mechanism Linked to PWS Focus of Early Study

News

The maternal SMCHD1 protein ensures genomic imprinting and the effective silencing of particular genes by protecting them from “gene-activating” proteins in the fertilized egg cell (or oocyte), according to a ... Read more

November 19, 2020November 19, 2020

US Trial Testing Harmony’s Pitolisant for Excessive Sleep Starts Recruiting Patients

News

A Phase 2 clinical trial testing pitolisant as a treatment for excessive daytime sleepiness (EDS) in Prader-Willi syndrome (PWS) has started recruiting patients at its Texas site and is ... Read more

November 17, 2020November 17, 2020

Latest NORD Webinar Offers Insights on Starting Nonprofit, Patient Registry

News, syndicated

Leaders in the U.S. rare disease community came together recently for a webinar to present helpful information on how to start a nonprofit and patient registry. They shared about ... Read more