News

People with Prader–Willi syndrome (PWS) who received growth hormone therapy had significantly higher vocabulary IQ scores compared with those who did not have the treatment, a study shows. The study also revealed that PWS patients whose disease is caused by maternal…

Imagine living your whole life with a painful disease so rare that only 25 others worldwide have what you have. And that you’re one of just six such people who’ve made it to adulthood. Neena Nizar doesn’t have to imagine. The 41-year-old English professor at Metro Community College in Elkhorn,…

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…