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Medix has submitted a new drug application to the Mexican food and drug administration — Comisión Federal para la Protección contra Riesgos Sanitarios, or COFEPRIS — seeking approval of tesofensine for the treatment of obesity. Tesofensine is one of two compounds that make up Tesomet, which Saniona…

Difficulties that people with Prader-Willi syndrome (PWS) have in adapting to their surroundings appear to be linked to psychiatric problems, generalized anxiety and hyperactivity, researchers report. Their study, “The Profiles and Correlates of Psychopathology in Adolescents and Adults with Williams, Fragile X and Prader-Willi Syndromes,” which also looked…

A genetic analysis of hibernating animals and people with Prader-Willi syndrome (PWS) has revealed more than 300 small genetic sequences that may regulate fat accumulation and obesity, a study reports. This discovery adds new knowledge on the underlying processes of fat buildup and reveals new clues on potential genetic factors…

Despite low muscle tone, young adults with Prader-Willi syndrome can do exercises involving resistance training and likely will benefit from greater muscle strength, a study suggests. The research, “Myokine levels after resistance exercise in young adults with Prader–Willi syndrome (PWS),” was published in the American Journal of Medical…

Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or so Americans with rare diseases and ensuring timely, affordable access to lifesaving treatments, the nation’s highest-ranking health official said. “We have to think about how our financing system can protect those with serious and rare illnesses.

Adults with Prader-Willi syndrome (PWS) have difficulty recognizing facial expressions, which may contribute to their poor social skills, a study reveals. The researchers found that people with PWS also show atypical, autistic-like features, depending on the specific genetic cause of the disease. These findings expose…

Telomeres — a marker of cell division and lifespan — were found to be shorter in young adults with Prader-Willi syndrome (PWS) than in healthy controls, which could be a sign of premature aging, a study reports. The study, “Evidence for accelerated biological ageing in young adults…

Early diagnosis and treatment of Prader-Willi syndrome may delay obesity and minimize the risk for associated complications, a study suggests. The study, “Early Diagnosis in Prader–Willi Syndrome Reduces Obesity and Associated Co-Morbidities,” was published in the journal Genes, and was led by researchers at the University…

The U.S. Food and Drug Administration (FDA) has granted fast track designation to LV-101 (intranasal carbetocin), a treatment candidate for people with Prader-Willi syndrome (PWS). This designation is granted to promising therapies intended to treat serious diseases with unmet medical needs. It aims to quicken treatment development by…

Rare disease-themed videos glowed on a large screen before an audience of people in wheelchairs, with crutches, and bearing oxygen tanks this Nov. 9 and 10 in San Francisco. Disorder: The Rare Disease Film Festival strives to eventually host a film about every one of the nearly 7,000 rare…