Author Archives: Marta Figueiredo

Researchers have identified a new SDCCAG8 gene mutation associated with Bardet-Biedl syndrome (BBS) in an Iranian family. The cases were reported in the study “A novel splice site mutation in the SDCCAG8 gene in an Iranian family with Bardet–Biedl syndrome,” published in the journal International…

The U.S. Food and Drug Administration (FDA) has accepted and given priority review to Vanda Pharmaceuticals’ request seeking the approval of Hetlioz (tasimelteon) as a treatment for people with Smith-Magenis syndrome (SMS), a rare genetic disorder characterized by sleeping problems and often associated with obesity. Sold in 20 mg…

Rhythm Pharmaceuticals’ setmelanotide promotes weight loss not only in adults, but also in children and adolescents with POMC deficiency and LEPR deficiency, two rare genetic obesity disorders, according to updated data from two Phase 3 clinical trials. “We are encouraged to see therapeutic activity in children and adolescents,” Murray…

A new weekly formulation of setmelanotide, Rhythm Pharmaceuticals’ lead treatment candidate for rare genetic obesity disorders, is as safe and effective in promoting weight loss as the daily formulation, interim data from a Phase 2 trial show. The findings, collected from people with obesity but without these genetic…

Mutations in two blood pressure-related genes — angiotensinogen (AGT) and angiotensin-converting enzyme (ACE) — may increase a person’s risk of being overweight or obese, according to a study of Tunisians. While previous studies in several ethnic populations have reported conflicting results, these findings support the existence of a…

The U.S. Food and Drug Administration (FDA) has accepted and given priority review to Rhythm Pharmaceuticals’ request that setmelanotide be approved to treat people with POMC deficiency and LEPR deficiency, two rare genetic obesity disorders. This priority status is expected to shorten setmelanotide’s regulatory review to…

Rhythm Pharmaceuticals is seeking approval from the U.S. Food and Drug Administration (FDA) for its lead candidate setmelanotide to treat POMC deficiency and LEPR deficiency, two rare genetic obesity disorders. Over the next two months, the FDA will determine whether Rhythm’s new drug application (NDA) is acceptable for filing…

The offspring of obese mothers are more likely to age faster, and to develop metabolic diseases such as diabetes and heart disease, a study in rats shows. While the health problems spanned the offsprings’ lifetimes, the researchers noted that “encouragingly, exercise by the offspring improves many of the poor offspring…