News

While there are few silver linings to the cloud created by COVID-19, the pandemic that has killed tens of thousands, hobbled economies worldwide and drove millions to quarantine in their homes, one may be a new appreciation of telemedicine. “If something good could come out of this crisis, it’s that…

The approach used by diazoxide choline controlled-release (DCCR), Soleno Therapeutics’ investigational oral therapy to reduce appetite in people with Prader-Willi syndrome (PWS), has shown promise in improving insulin sensitivity and reducing body fat in several animal models of obesity linked to excessive eating. The findings were…

The National Organization for Rare Disorders (NORD) has opened a financial assistance program for people in the rare disease community who are affected by the COVID-19 pandemic in the U.S. Called the NORD COVID-19 Critical Relief Program, the effort will provide up to $1,000 annually to those eligible…

Children with Prader-Willi syndrome (PWS) struggle to express adequate facial and bodily emotions, which is linked to difficulties adapting socially, a study suggests. The study, “Equivocal expression of emotions in children with Prader-Willi syndrome: what are the consequences for emotional abilities and social adjustment?,” was published in the…

First, the bad news: If you’re one of the 30 million or so Americans with a rare disease, you probably have lower immunity to the novel coronavirus than most people. Now, the good news: You already know how to face loneliness and adversity — qualities that make you far stronger…

Millendo Therapeutics‘ treatment candidate livoletide failed to significantly reduce hunger and improve food-related behaviors in people with Prader-Willi syndrome (PWS) who are taking part in the pivotal ZEPHYR Phase 2b/3 clinical trial. Also, the treatment did not improve other measures, such as body fat, waist circumference,…

Parents and teachers attribute different severity to behavioral problems of children with Prader–Willi syndrome (PWS), according to a recent study. Although parents see food-related issues, such as repetitive speech and compulsive behavior as being more severe, teachers report that the impact of such problems on daily activities is higher. The study,…

The Living Rare, Living Stronger Patient and Family Forum, originally set for May 14–16 in Cleveland, Ohio, has been postponed until July 18–20 because of the coronavirus disease COVID-19 pandemic. The event’s sponsor, the National Organization for Rare Disorders (NORD),…

Altered gene expression underlies impaired function of brain areas regulating vision and motor control, which may be associated with behavioral issues in people with Prader-Willi syndrome (PWS) and other disorders, a study suggests. The study, “Central neurogenetic signatures of the visuomotor integration system,” was published…

Even with the coronavirus pandemic ravaging Europe and much of the world, patient advocate Lucia Monaco, PhD, of Italy remains confident that the Paris-based nonprofit she chairs will see the approval of 1,000 new rare disease therapies by 2027. That group, the International Rare Diseases Research Consortium (IRDiRC) —…