Forest Ray PhD,  —

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…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Through the end of the year, the Prader-Willi Syndrome Association USA (PWSA USA) is offering 24-hour family support services to everyone in the Prader-Willi syndrome (PWS) community. The association’s family support team will provide emergency access to information and support for urgent medical and behavioral emergencies. Those in need…

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…

Lallemand Health Solutions has partnered with the Prader-Willi Syndrome Association (PWSA) to support Running for Research, an initiative to raise funds for PWS 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…

Same But Different, a U.K. nonprofit that uses the arts to bring communities together, is holding a calendar photography competition to raise awareness for rare diseases. Under the theme “A Glimmer of Hope,” the competition is a means to “visually express the hope that exists for people affected by…

The Foundation for Prader-Willi Research (FPWR) is hosting a video competition to help spread awareness of Prader-Willi syndrome (PWS) and raise funds for research. The FPWR is seeking inspiring stories from the PWS community in the form of short videos, of one-to-five minutes in length. Possible…

Raremark, an online rare disease patient community, has launched a digital platform called Xperiome, aimed at streamlining the search for new medicines for rare disorders and incorporating more patient input into research. The goal is to help the pharmaceutical industry deliver innovative new therapies faster and smarter, the…