News

People with Prader-Willi syndrome (PWS) are aware of the disease, but not of its social consequences, a small single-site study has found. These findings may have important clinical implications for care as “patients believe they are capable of independent living when they are actually not,” the researchers wrote. The…

Those who wish to gain practical tools for living optimally with rare diseases are encouraged to attend the annual Living Rare Living Stronger Patient and Family Forum, hosted by the National Organization for Rare Disorders (NORD) and set this year for June 26-27. The conference brings together patients,…

In adults with Prader-Willi syndrome (PWS), use of growth hormone (GH) treatment may improve the quality of sleep with no significant negative effects on breathing, such as obstructive sleep apnea, a Scandinavian study suggested. The study, “Effects of Growth Hormone treatment on sleep-related parameters in adults with Prader-Willi…

The goal of the Foundation for Prader-Willi Research (FPWR) is to fund and support studies that will help “to eliminate the challenges” faced by people with the rare genetic disease, its leadership said in a recent webinar highlighting the FPWR’s ongoing studies and other projects. To that end, Susan…

Eurordis opened a campaign, called Rare 2030 Action, that is seeking to establish a European action plan for rare diseases to ensure that none of the 30 million people in Europe living with rare diseases are left behind by the start of a new decade. As part of…

The genetic profile of children with Prader-Willi syndrome (PWS) correlates with their age at diagnosis and at the start of growth hormone treatment, as well as with pre-treatment levels of insulin-like growth factor 1 (IGF1) — a hormone that promotes…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000in  grants, totaling up…

Editor’s Note: This is the second of two articles looking at DCCR, Soleno Therapeutics’ investigational treatment, and the DESTINY-PWS trial. The first can be found here.  Likely next steps for diazoxide choline controlled release (DCCR) tablets, a potential therapy for Prader-Willi syndrome (PWS), remain to be known,…

Diazoxide choline controlled release (DCCR) tablets are an investigational therapy for Prader-Willi syndrome (PWS) that, according to data from a Phase 3 clinical trial and linked extension study, may be the first to control the disorder’s hallmark symptom: hyperphagia, or an insatiable hunger. But the COVID-19 pandemic that…

Lockdown during the COVID‐19 pandemic led to weight reduction among half of adults with Prader-Willy syndrome (PWS) in a French study, data show. Weight loss typically was seen in patients with higher physical activity and better eating habits. Moreover, these findings likely reflect changes in lifestyle behaviors and…