Aripiprazole, a treatment used for schizophrenia and other brain disorders, may potentially worsen behavioral problems in young people with Prader-Willi syndrome (PWS), according to a small study. Symptoms such as aggressiveness, irritability, and impulsiveness intensified in six people with PWS who received aripiprazole, but eased once they stopped taking…
News
There may be changes to how the “feel-good” hormone oxytocin works in patients with Prader-Willi syndrome (PWS) in general, and in those with psychosis, according to findings from a small study in Germany. Researchers found that OXTR, the gene that codes for the oxytocin receptor, is not as methylated in…
Sleep disturbances in people with Prader-Willi syndrome (PWS) are linked with psychosis-risk symptoms, a study reports. Psychosis refers to disturbed thoughts and perceptions that may make it difficult to understand what is real and what’s not. The findings also indicate neurobehavioral symptoms and cognition can be analyzed remotely in…
A total of 30 patient advocacy groups working to further understanding and the needs of people living with rare diseases have been selected to receive a Horizon Therapeutics‘ #RAREis Global Advocate Grant. Winners of this year’s inaugural awards are spread across nine countries and represent a total of 29 rare…
The first patient has been randomly assigned to a dosing group in the pivotal Phase 2/3 SCOUT-015 trial, which will evaluate the safety and efficacy of RAD011, an investigational cannabidiol oral treatment, in people with Prader-Willi syndrome (PWS). SCOUT-015 (NCT05098509) is designed to test the effects of…
Note: This story was updated July 13, 2022, to correct the name of Rare-X’s CEO Charlene Son Rigby. Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world.
Early treatment with growth hormone — before 1 year of age — was associated with better body composition and cognitive function after eight years in children with Prader-Willi syndrome (PWS), according to a recent Dutch study. The findings support additional research favoring early treatment with growth hormone for…
Testosterone injections are well-tolerated and normalize blood levels of the hormone, but they may raise red blood cell levels excessively in adult men with Prader-Willi syndrome (PWS), according to a recent study. The study, “Single-center real-life experience with testosterone treatment in adult men with Prader-Willi syndrome,”…
A special low-calorie diet and appetite suppressing medications — either alone or together — led to substantial weight loss for obese people with Prader-Willi syndrome (PWS), a small study showed. However, many patients struggled to adhere to the treatment regimens and eventually regained the weight, the researchers reported. A very…
The National Organization for Rare Disorders’ (NORD) “Living Rare, Living Stronger Patient and Family Forum” is back in person on June 26 for a day of learning and networking in Cleveland, Ohio. The event, which brings together the rare disease community, will take place at the InterContinental Cleveland Conference…
Recent Posts
- 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
- Adults with PWS need lifelong support in daily activities, study finds
- Celebrating a milestone in life with Prader-Willi syndrome
- New technique ‘wakes up’ silent genes in Prader-Willi in lab testing