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…
News
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…
Recent Posts
- Advocating for your child with Prader-Willi syndrome is a lifelong endeavor
- Collecting pop tabs is a small act that makes a big impact
- Safety concerns over PWS therapy ARD-101 pause global clinical trial
- Starting growth hormone before age 2 does not harm PWS sleep
- I’m hopeful a new treatment will benefit my son with Prader-Willi syndrome
- Palatin to test 2 obesity-targeting drugs for PWS in clinical trials this year
- Still-recruiting ARD-101 trial for PWS lowers US enrollment age to 7
- For rare disease families, February is a month of both love and awareness
- Muscle, bone signaling imbalances found in non-obese PWS children
- Most PWS patients meet nutrient targets but struggle with calorie control