News

Pandemic Won’t Stop Rare Disease Day on Feb. 28

Scores of virtual events are afoot around the world to mark Rare Disease Day 2021 on Feb. 28. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…

Antidepressant Sertraline May Help Control Temper Outbursts

The antidepressant sertraline may help control temper outbursts in people with Prader-Willi syndrome (PWS), a small study suggests. Among 13 patients who completed six months of treatment with sertraline, the researchers reported a greater than 90% decrease in the frequency of temper outbursts. Treatment over six months also…

NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns

While progress was made last year on newborn screening and other policy issues critical to rare disease patients, a “State Report Card” argues that many concerns — notably out-of-pocket costs for prescription medicines and access to affordable comprehensive care — still need attention. Those were the findings of the…

Growth Hormone Therapy Appears Safe and Helpful for PWS Adults

Growth hormone therapy, a common treatment for children with Prader-Willi syndrome (PWS),  is also safe and effective at increasing lean muscle mass and lowering body fat in adults with this disease, a review study reports. More work is needed, however, to confirm the therapy’s effects on body composition, as well…

Higher Risk of Autism Spectrum Disorder Found in PWS Children

More than 70% of children and adolescents with Prader-Willi syndrome (PWS) have a high risk of developing autism spectrum disorder (ASD), according to a study in Chinese patients. The findings also indicated that individuals who were shorter, had excess weight, and experienced obstructive sleep apnea — a condition in…