Data from an ongoing Phase 2a clinical trial showed that Tesomet (tesofensine/metoprolol) is safe and well-tolerated by adolescents with Prader-Willi syndrome (PWS). Eight of the nine adolescents included in the trial have enrolled in an open-label extension (OE) study, where they will proceed with Tesomet treatment for an additional…
The world’s biggest gathering of rare disease researchers, patient groups, pharmaceutical executives, and government officials is planned for April 10–12 in a Washington, D.C., suburb. Some 1,200 people have already registered to attend the World Orphan Drug Congress (WODC) USA 2019, set to take place at the Gaylord National Harbor…
About 100 scientists, researchers, pharmaceutical executives, and others will converge on Austria’s capital city early next month for the 2nd International Congress on Advanced Treatments in Rare Diseases. The March 4-5 meeting, to take place at the Hilton Am Stadtpark Vienna, features 27 speakers on a variety of disorders…
A low-intensity training program improved hand function in adults with Prader-Willi syndrome (PWS), according to results of a pilot study in six patients. The research, “Hand strength and dexterity in patients with Prader-Willi syndrome: a pilot intervention study,” was published in the Journal of International Medical…
With careful multidisciplinary evaluation and psychosocial support, adolescents with Prader-Willi syndrome (PWS) with severe obesity can undergo bariatric surgery with successful results, a case report suggests. The study, “Laparoscopic sleeve gastrectomy in an adolescent with Prader-Willi syndrome: psychosocial implications,” was published in the journal Nutrition. PWS is the most…
Scores of events are afoot worldwide to mark Feb. 28, Rare Disease Day 2019. The activities aim to raise awareness about rare diseases and the millions of people — estimates run as high as 350 million — they are thought to impact. Across countries, patients, caregivers and advocates will paint faces, wear…
Involuntary reflexes such as being startled are abnormal in Prader-Willi Syndrome (PWS) patients, and lower startle response to food images may explain their eating habits, researchers from the University of Kansas Medical Center report. The study, “Startle Response Analysis of Food-image Processing in Prader-Willi Syndrome” was published in…
Disgusting food scenes failed to activate primitive limbic structures in the brains of people with Prader-Willi syndrome (PWS), but cortical responses — where conscious sensations are processed — were almost normal, a study reports. The study, “Lack of Response to Disgusting Food in the Hypothalamus and Related Structures in Prader…
Adults with Prader-Willi syndrome (PWS) show prematurely aged brains, being on average 8.74 years older than their chronological age, according to a neuroimaging study. This was not associated with body mass index (BMI), use of hormonal or psychotropic medications, severity of repetitive or disruptive behaviors, or intelligence quotient (IQ). The study,…
G71.01 is, literally, the code for Duchenne muscular dystrophy. Q93.51 stands for Angelman syndrome, and G40.419 covers generalized and treatment-resistant epilepsies, which groups like Orphanet and the American Epilepsy Society define as including Dravet syndrome. All three designations are among some 70,000 diseases listed in the latest…
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