News

Certain risk factors — such as older age, obesity, metabolic syndrome, cardiovascular disease, and kidney failure — increase the likelihood of blood clots in patients with Prader-Willi syndrome (PWS), according to results of a national U.S. survey. The study, “Venous Thromboembolism in Prader–Willi Syndrome: A Questionnaire…

Inhibiting an enzyme involved in Prader Willi syndrome (PWS) can help rescue the activity of specific genes in neurons, and halt disease, a lab study suggests. The study “Small molecule inhibitors of G9a reactivate the maternal PWS genes in Prader-Willi-Syndrome patient derived neural stem cells and…

A potential new treatment for Prader-Willi syndrome (PWS) will be tested in the large, international clinical trial ZEPHYR, in a partnership between Millendo Therapeutics and several research centers, including The University of Alabama at Birmingham…

Oklahoma suffers more tornadoes than any other state, has the highest per-capita rate of women in U.S. prisons, ranks second in the number of teen births per 100,000 teenage girls, and has the nation’s third-highest rate of uninsured residents — with 13.9% of all Oklahomans lacking health coverage. As if…

Screening newborns for genetic diseases with treatments that can prevent crippling or deadly progression, especially for rare disorders, has a ways to go in the United States. No state today tests for all 35 disorders recommended under a federal screening panel, and even in those that come close, rare…

The ongoing DESTINY PWS Phase 3 trial is halfway to its target enrollment of patients with Prader-Willi syndrome (PWS) to evaluate the impact of once-daily treatment with diazoxide choline controlled-release (DCCR) tablets. Importantly, more than 90% of the patients who have completed the study so far have chosen to…

There is a wide range of long-term testicular function and cell-level appearance in males with Prader-Willi syndrome (PWS), a new study shows. The study, “Gonadal function and testicular histology in males with Prader‐Willi syndrome,” was published in the journal Endocrinology, Diabetes, & Metabolism. It is quite common…

Two organizations that advocate for people with Prader-Willi syndrome have teamed up with Boston-based Zafgen to conduct an unprecedented natural history study of the rare neurodevelopmental disorder. PATH for PWS is supported by both the Foundation for Prader-Willi Research (FPWR), headquartered in Walnut, California, and the…

Treatment with once daily guanfacine extended-release tablets — used to treat attention deficit hyperactivity disorder (ADHD) — may help manage behavioral symptoms, such as aggression and self-injurious behavior, associated with Prader-Willi syndrome (PWS). The findings were reported in the study “Guanfacine Extended Release for…

A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…