News

Rhythm Pharmaceuticals has identified 31 additional genes whose mutations may be amenable to setmelanotide, its U.S.-approved therapy for genetic obesity disorders linked to the melanocortin-4 receptor (MC4R) pathway. This discovery is the result of Rhythm’s sequencing efforts and precision approach that has been recently validated in a Phase…

The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide regulatory agencies are expected to pose little risk to the rare disease community, including to patients with compromised immune systems or those participating in gene therapy studies. That was the message of a recent…

Three months of treatment with setmelanotide, an approved therapy for genetic obesity disorders, led to clinically meaningful body weight loss in adolescents and adults with additional diseases linked to the melanocortin-4 receptor (MC4R) pathway, data show. These interim data, from a Phase 2 basket trial, highlight setmelanotide’s potential…

A large-scale study spanning six decades found that people in Norway who are least genetically predisposed to obesity are relatively protected from it, while those most predisposed are at risk of both obesity and severe obesity. These findings suggested that interactions between genes and an…

Caring for a loved one with a rare disease, especially during these uncertain times, demands significant time, attention, patience, and dedication. To help meet that need, the National Organization for Rare Disorders (NORD)’s Rare Caregiver Respite Program may be a helpful resource. The program seeks to give a…

The Mediterranean diet reduces obesity-related metrics in adolescents, but it is not as effective in males who have a high genetic risk for obesity, a study has found. In female adolescents, the diet significantly lowered body mass index (BMI), waist circumference, and metabolic syndrome, even in…

Deleting a gene called Trim28 in mice led to alterations in fat storage and metabolism, especially in female mice, who became significantly heavier, a study reported. These findings demonstrate Trim28‘s role in genetic obesity and show that the…

Genetic risk factors underlying childhood obesity differ from those in adulthood, a large-scale analysis found. Better understanding of such risk factors could further the study of childhood obesity in relation to health later in life.  The study, “Separating the genetics…

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. …