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The U.S. Food and Drug Administration (FDA) has provided some recommendations to Saniona regarding the development of Tesomet to treat Prader-Willi syndrome (PWS). In particular, the agency is advising the company to conduct a Phase 3 trial to follow the planned Phase 2b clinical trial, as well…
Same But Different, a nonprofit U.K. group that uses art for social change, is inviting people to choose their favorite photographs in a calendar contest to heighten awareness of rare diseases, including amyotrophic lateral sclerosis (ALS). The organization’s panel of judges has pared the number of contest submissions…
Daily treatment with diazoxide choline controlled release (DCCR) leads to sustained reductions in excessive appetite and other symptoms of Prader-Willi syndrome (PWS), according to updated findings from a Phase 3 trial. “The results from this program continue to demonstrate DCCR’s beneficial impact on hyperphagia [excessive appetite], the…
An online survey from Trinity College Dublin is seeking input from the parents and caregivers of young people with Prader-Willi syndrome about the impact of the COVID-19 pandemic on the children’s mental health and behavior. Besides Prader-Willi, the scientists are also studying patients with autism, as well…
It took one year for Dona Krystosek to get a diagnosis for her son, Levi, after he was born. The family received three misdiagnoses of fatal diseases until they found out Levi has Jansen’s metaphyseal chondrodysplasia — an extremely rare form of dwarfism.  “The hardest…
Levels of uric acid in the blood are lower in adults with Prader-Willi syndrome (PWS) who are obese than in people without PWS but who also are obese, a study shows. Fat-free body mass was the strongest predictor of uric acid levels. Researchers…
Text messages may help to track changes in the weight and body mass index (BMI) of people with Prader-Willi syndrome (PWS), an observational study reported. Most study patients were “highly compliant” with using this mobile technology, and it might help in clinical trials of potential treatments or with…
Japanese infants with Prader-Willi syndrome (PWS) have lower levels of the thyroid hormone thyroxine compared with healthy infants, according to a new study. However, the levels of thyroxine, also known as free T4 (FT4), increased with age, with no differences detected between toddlers with or without PWS,…
Lallemand Health Solutions has partnered with the Prader-Willi Syndrome Association (PWSA) to support Running for Research, an initiative to raise funds for PWSÂ research at the University of Florida (UF). Running for Research has set a fundraising goal of at least $40,000, and will take place…
Inversago Pharma has obtained $35 million in financing to advance the development of therapies for disorders such as Prader-Willi syndrome (PWS). The investment will support studies of INV-101, the company’s lead compound, and other selected compounds for the treatment of metabolic and fibrotic (scarring) disorders. INV-101 is an…
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