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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.

A small study exploring gene alterations related to Prader-Willi syndrome (PWS) suggests that subtypes of the disorder have different genetic profiles that may affect response to psychotropic treatments and potential side effects. Due to its small size, only trends rather than statistically significant differences were…

Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place on March 1, will feature panel discussions, patient stories, research updates, TED-style talks, and a presentation by a Nobel laureate recently recognized for her work on a gene editing tool. The free, virtual…

People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of an international survey conducted by Eurordis-Rare Diseases Europe. Indeed, rare disease patients overall give their healthcare experience a medium-low rating, of 2.5 on a scale of 1 to 5,…