News

Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…

Saniona will continue to develop Tesomet (tesofensine/metoprolol) as a potential therapy for Prader-Willi syndrome (PWS), with results from a Phase 2a clinical study expected to be reported soon. The company has completed a rights issue in which it sold some of Saniona’s shares, resulting in gross revenue of SEK…

The upcoming Research Symposium and Family Conference, hosted by the Foundation for Prader-Willi Research (FPWR), will offer the latest in research studies and promising treatments, and the opportunity to connect with the Prader-Willi syndrome (PWS) community. Held in conjunction with the PWS Research Symposium, the annual event will…

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…