People with rare disorders have a worse healthcare experience than those affected by chronic diseases, according to the results of…
Articles by Marta Figueiredo
Suppressing the activity of a cellular transporter called multidrug resistance protein 4 (Mrp4) results in more fat tissue, less energy…
Rhythm Pharmaceuticals has identified 31 additional genes whose mutations may be amenable to setmelanotide, its U.S.-approved therapy for…
Three months of treatment with setmelanotide, an approved therapy for genetic obesity disorders, led to clinically meaningful body weight…
One year of treatment with setmelanotide was safe and resulted in a significant drop in body weight and hunger…
Hetlioz (tasimelteon) has been approved in the U.S. to treat nighttime sleeping problems in people, ages 3 and older,…
Two mutations in or near the LEP gene resulting in lower levels of the hunger-reducing hormone leptin…
The U.S. Food and Drug Administration (FDA) has approved Imcivree (setmelanotide) as the first-ever treatment for people, 6…
The first wave of COVID-19Â in Europe severely disrupted access to care and raised stress and anxiety in people with…
People with Bardet-Biedl syndrome (BBS) have nearly two times as many rare secondary mutations in BBS-associated genes than their…