Results of a Phase 2 clinical trial testing diazoxide chloride controlled release (DCCR) in people with Prader-Willi syndrome (PWS) showed the investigational therapy can address some unmet needs in this patient population — namely hyperphagia, or excessive appetite, and aggressive behavior. The data were published in the journal …
Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…
More than 800 people are expected to converge on Orlando, Florida, in late October for the 35th National Convention of the Prader-Willi Syndrome Association (PWSA). The event, to take place over a four-day period at the Caribe Royale Hotel, is divided into three segments: a two-day medical and scientific…
Saniona announced that adolescents with Prader-Willi syndrome (PWS) showed improvements in weight, body mass index (BMI), and hyperphagia score — measuring appetite reduction — when treated with the investigational therapy…
The deficit in social cognitive abilities among young children with Prader-Willi syndrome (PWS) starts at an early age — and varies with the genetic subtype, a study found. Children with PWS who inherit two chromosomes 15 from their mother showed manifestations similar to those of autism spectrum…
Newborns with Prader-Willi syndrome often have a lower birth weight and can be shorter than newborns who do not have this rare disease, a study conducted in Italy reports. Recognition of these body features may help clinicians in diagnosing the syndrome earlier, and in beginning suitable support and treatment measures…
Rare diseases deeply affect not only the children who experience them, but also their healthy brothers and sisters, as their parents can attest. Two entries in November’s “Disorder: The Rare Disease Film Festival” will focus on what siblings go through, according to the San Francisco festival’s co-founder,…
Both growth friendly surgery (GFS) and serial cast correction (SCC) were found to help resolve early-onset scoliosis, and to improve spine structure, in children with Prader-Willi syndrome (PWS), according to a two-year study. The findings from the study, “Growth Friendly Surgery and Serial Cast…
Developing gene therapies for rare diseases is one thing. Creating gene-edited “designer babies” is quite another. German legal expert Timo Minssen outlined the potentially explosive ethical landmines surrounding such issues during a recent talk at the New York Genome Center. Minssen directs the Center for Advanced Studies in…
Saniona will continue to develop Tesomet (tesofensine/metoprolol) as a potential therapy for Prader-Willi syndrome (PWS), with results from a Phase 2a clinical study expected to be reported soon. The company has completed a rights issue in which it sold some of Saniona’s shares, resulting in gross revenue of SEK…
Get regular updates to your inbox.