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The National Organization for Rare Disorders (NORD) is seeking individuals willing to share real-life experiences with rare diseases to speak at its upcoming virtual Living Rare, Living Stronger NORD Patient and Family Forum. The interactive, patient-focused forum will be held online June 26-27. The deadline to apply for…

In a $100 million deal, Rhythm Pharmaceuticals is selling its rare pediatric disease priority review voucher (PRV), granted by the U.S. Food and Drug Administration (FDA) with the approval of Imcivree (setmelanotide) to treat three rare genetic obesity disorders. …

One year of treatment with setmelanotide was safe and resulted in a significant drop in body weight and hunger in Bardet-Biedl syndrome (BBS) patients, ages 12 years and older, with moderate to severe obesity, according to top-line data from a Phase 3 clinical trial. “We are pleased with…

Genetic screening in fruit flies is a relatively simple, cost-effective, and rapid process with the potential to increase understanding of genetic obesity development in humans. Specifically, the fruit fly is a workable model organism for high-throughput screening of candidate genes associated with eating behavior and obesity in humans, according to…

Genetic susceptibility toward obesity has little impact on whether lifestyle interventions aimed at weight loss are successful in children who are overweight or obese, a new study suggests. The study, “Obesity Genes and Weight Loss During Lifestyle Intervention in Children With Obesity,” was published in the journal…

Having a higher genetic likelihood for greater body mass index (BMI), a measure of body fat, increases the risk for rheumatoid arthritis (RA), according to an analysis of data of more than 700,000 people. The study, “Obesity‐related traits and the development of rheumatoid arthritis — evidence…

Adult survivors of childhood acute lymphoblastic leukemia (ALL), who are at high risk for obesity, exhibit alterations in genetic variants associated with body mass index (BMI) — a measure of body fat based on height and weight — following cranial radiation therapy (CRT), a study reports.

Hetlioz (tasimelteon) has been approved in the U.S. to treat nighttime sleeping problems in people, ages 3 and older, with Smith-Magenis syndrome (SMS), a rare genetic disorder also linked to obesity. The 20 mg capsule formulation — already approved to treat a sleep disorder called non-24-hour sleep-wake disorder —…

Two mutations in or near the LEP gene resulting in lower levels of the hunger-reducing hormone leptin are associated significantly with a higher body mass index (BMI) in early childhood, but not in adulthood, according to a large, international genetic study. These results suggest an age-dependent link…

To empower and equip members of the rare disease community to engage state leaders in matters of importance to patients and their families, the National Organization for Rare Disorders (NORD) has launched an initiative across the U.S. Its goal is to establish a Rare Disease Advisory Council (RDAC)…