Prader-Willi News

September 17, 2021September 17, 2021

New Institute Aims to Leave No Rare Disease Patient Behind

Prader-Willi News

A newly launched non-profit institute is seeking to advance research, and the development of new therapies, for people with rare diseases — a patient community with some of the largest ... Read more

September 16, 2021September 16, 2021

Dosing Begins in Phase 1 Trial of INV-102, Potential PWS Therapy

Prader-Willi News

The first healthy volunteers were dosed in a Phase 1 clinical trial evaluating Inversago Pharma‘s INV-102, a potential therapy for Prader-Willi syndrome (PWS) and other metabolic disorders. According to a ... Read more

September 14, 2021September 14, 2021

PWS Community Asked to Take Part in Nov. 4 FDA Virtual Meeting on LV-001

Prader-Willi News

The U.S. Food and Drug Administration (FDA) is holding a public advisory committee meeting regarding LV-101 (intranasal carbetocin) as a potential treatment of distress behaviors and insatiable hunger in Prader-Willi ... Read more

September 9, 2021September 10, 2021

Results of DCCR Extension Study Support FDA Application, Soleno Says

COVID-19 News, Prader-Willi News

One year of treatment with Soleno Therapeutics’ investigational oral therapy diazoxide choline controlled release, known as DCCR, significantly reduced excessive appetite and disease-related behaviors in Prader-Willi syndrome (PWS) patients, according ... Read more

September 8, 2021September 8, 2021

Group Focuses on Rare Disease Clinical Trial Participation

Prader-Willi News

Participation in clinical trials exposes rare disease patients to financial, physical, and emotional pressures, according to the results of a patient focus group series. “Rare disease trial participants are running ... Read more

September 7, 2021September 7, 2021

Study Links Pituitary Gland Changes in PWS to Behavioral Symptoms

Prader-Willi News

People with Prader-Willi syndrome (PWS) have smaller pituitary glands than their typically-developing peers, according to data from a small study. The findings also suggest that altered pituitary size is associated ... Read more

September 2, 2021September 3, 2021

Early Screening, Diagnosis May Reduce High Annual Disease Costs in PWS

Prader-Willi News

The mean total annual costs of raising a child with Prader-Willi Syndrome (PWS) — an estimate that takes into account services use, impact on parental income, and care arrangements — ... Read more

August 31, 2021August 31, 2021

‘Running for Rare’: NORD a Charity Partner of 2021 NYC Marathon

Prader-Willi News

The National Organization for Rare Disorders, known as NORD, was named an official charity partner of the 2021 TCS New York City Marathon, which will be held Nov. 7 both ... Read more

August 27, 2021August 27, 2021

‘Not Alone in the Dark’: New Guide for, by Caregivers of Children With Rare Diseases

Prader-Willi News

The National Alliance for Caregiving, in partnership with Global Genes, has issued a free guidebook, available online, that offers resources and support for caregivers of children with rare diseases. “The ... Read more

August 26, 2021August 26, 2021

High-resolution Imaging of Entire Human Genome on the Horizon

Prader-Willi News

An $11.2-million, five-year grant has been awarded by the Centers of Excellence in Genomic Science of the National Institutes of Health (NIH) to uncover high-resolution details of the entire human ... Read more

August 24, 2021August 24, 2021

Early Screening, Treatment of Vision Problems Important for PWS Patients

Prader-Willi News

Vision problems such as lazy eye, farsightedness, and strabismus — when the eyes don’t line up in the same direction — are more common in people with Prader-Willi syndrome (PWS) than ... Read more

August 19, 2021August 19, 2021

Study: PWS Doesn’t Increase Risk of Severe COVID-19

COVID-19 News, Prader-Willi News

Children and adults with Prader-Willi syndrome (PWS) are not at higher risk of developing a severe COVID-19 infection, according to results from a survey involving more than 500 patients in France. These ... Read more

August 18, 2021August 18, 2021

Register Now for Global Genes’ RARE Patient Advocacy Summit

Prader-Willi News

Registration is now open for Global Genes‘ 2021 RARE Patient Advocacy Summit. This year’s hybrid event will be livestreamed from California Sept. 27-29, and some seats also are available for attending ... Read more

August 17, 2021August 17, 2021

Teenager’s Response Supports Pitolisant, Now in Phase 2 Trial, in Easing PWS

Prader-Willi News

Pitolisant, a treatment for narcolepsy, should be considered for people with Prader-Willi syndrome (PWS) whose response to psychotropic medications is less than optimal, a case report of a 15-year-old patient ... Read more

August 12, 2021August 12, 2021

Hunger Hormone Ghrelin May Predict Early Onset of Scoliosis in PWS Children

Prader-Willi News

The hunger hormone ghrelin, when measured in infancy, may be a biomarker for predicting the early onset of scoliosis — a sideways curvature of the spine that looks like the ... Read more