Rhythm to present new data from ROAD testing program at ESPE 2023

Rhythm Pharmaceuticals announced four oral presentations at the 61st Annual European Society for Paediatric Endocrinology Meeting being held September 21-23, 2023 in The Hague, Netherlands. Andrea Haqq, M.D., Division of Pediatric Endocrinology, University of Alberta in Canada, and Jesus Argente M.D., Ph.D., Department of Pediatrics and Pediatric Endocrinology, Universidad Autonoma de Madrid in Spain, delivered an oral presentation on the effects of setmelanotide on the metabolic syndrome severity score based on body mass index score in pediatric patients with Bardet-Biedl syndrome. In an oral presentation titled, “Impact of Setmelanotide on Metabolic Syndrome Risk in Pediatric Patients with POMC and LEPR Deficiency,” researchers led by Martin Wabitsch, M.D., Ph.D., Department of Pediatrics and Adolescent Medicine, University of Ulm in Germany, presented new findings regarding setmelanotide treatment in pediatric patients with pro-opiomelanocortin or leptin receptor deficiency. In an oral presentation titled, “Frequency of MC4R Pathway Variants in a European Cohort of Individuals with Early-Onset Severe Obesity,” researchers led by Anthony Goldstone, M.D., Ph.D., Department of Brain Sciences, Imperial College London, assessed a large European-based cohort of individuals with early-onset severe obesity, finding that 20.4% of individuals sequenced carried a variant in one or more of 11 specific MC4R pathway-related genes, including 5.0% of individuals with variants classified as pathogenic, likely pathogenic or suspected pathogenic. “Frequency of Obesity-Related Gene Variants in a European Population with Early-Onset, Severe Obesity,” as presented orally by Jesus Argente M.D., Ph.D., Department of Pediatrics and Pediatric Endocrinology, Universidad Autonoma de Madrid in Spain, shows that among individuals with early-onset, severe obesity, approximately 31.4% carried variants believed to be associated with obesity. In addition, Rhythm is hosting a satellite symposium at ESPE 2023, titled, “Hyperphagia and early-onset, severe obesity: The role of precision medicine in the treatment of leptin melanocortin-4 receptor pathway diseases.”