News

Consumption of a high-protein diet shifts muscle cells’ metabolism to use fats rather than carbohydrates as an energy source in children and young adults with Prader-Willi syndrome (PWS), a pilot study in Canada suggests. The findings support the need for more studies to assess the implications of different types…

Specific genetic subtypes and growth hormone use both influence the physical characteristics, such as head shape and eye appearance, of people with Prader-Willi syndrome (PWS), a large study indicates. The study, “Influence of Molecular Classes and Growth Hormone Treatment on Growth and Dysmorphology in Prader‐Willi Syndrome:…

Adults with Prader-Willi syndrome (PWS) have a unique gut bacteria composition, characterized by low levels of probiotic species and a high number of other microbes whose potential health and metabolic implications are still unknown, a study has found. The findings indicate that people with PWS may benefit from probiotic…

The U.S. Food and Drug Administration (FDA) has told Soleno Therapeutics that an additional clinical trial “will be necessary” to support any future application seeking approval of diazoxide choline controlled release (DCCR) tablets for people with Prader-Willi syndrome (PWS), the company said. Soleno had submitted an updated analysis…

A $200,000 Comcast NBCUniversal grant has been awarded to The Arc to increase access to educational resources for students of color and economically disadvantaged youngsters with intellectual and developmental disabilities (IDD), which can include Prader-Willi syndrome.

Many diseases have their own awareness color — breast cancer is pink, muscular dystrophy is green, and AIDS is red, for example — but what’s the significance of pink, green, blue, and purple lights side-by-side? These are the colors most often used to represent Rare Disease Day. This…

Tesomet, a potential treatment for Prader-Willi syndrome (PWS), has been granted orphan drug status by the U.S. Food and Drug Administration (FDA). Saniona, the therapy’s maker, now plans to initiate a Phase 2b study of Tesomet in the first half of this year. “Receiving orphan drug designation…

Adults with Prader-Willi syndrome (PWS) have greater mental rigidity than those with matching intellectual disability, but without the disease, and those with normal intellectual level, according to a study from France. These findings support the presence of PWS-specific mental flexibility deficits, over and above intellectual problems, the researchers said.

Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a young age, he also learned how to turn those trials into triumphs. At age 9, a stroke due to his disease left him paralyzed…

Soleno Therapeutics has partnered with Vanderbilt University to develop therapies that work as ATP-dependent potassium channel activators for rare diseases, similar to the diazoxide choline controlled release (DCCR) tablets the company thinks might treat Prader-Willi syndrome (PWS). ATP-dependent potassium (or KATP) channels are proteins present in multiple body tissues.