News

Retrophin and the U.S. subsidiary of Britain’s Horizon Pharma will each donate $3 million over a six-year period to the Rare Disease Institute (RDI) at Children’s National Health System in Washington, D.C., helping it to strengthen care available and expand as a “center of excellence” for rare…

In recognition of Rare Disease Day 2018, Bionews Services — which publishes this website — will attend and report on three relevant conferences in the U.S. dealing with policies and programs of importance to patients and their families. The three are among 50 events in 32 states…

Soleno Therapeutics and the U.S. Food and Drug Administration recently held a meeting to mark the end of a successful Phase 2 clinical trial evaluating diazoxide choline controlled-release (DCCR) for the treatment of Prader-Willi syndrome (PWS). DCCR is an extended-release, crystalline salt formulation of diazoxide designed to…

Long-term use of growth hormone therapy in young adults with Prader-Willi syndrome (PWS) fails to prevent bone deterioration. But a combination of growth hormone therapy and sex hormone therapy may have therapeutic potential for PWS patients, a new study suggests. The findings were reported in a study titled, “…

In people with Prader-Willi syndrome, the severity of aberrant behaviors declines with age, a phenomenon that starts at around age 30, according to Japanese researchers. Food-related behaviors, however, are maintained and don’t seem to change as patients grow older. The study, “Aberrant, autistic, and food-related behaviors…

Saniona recently reported encouraging results from its exploratory Phase 2a clinical trial evaluating Tesomet (tesofensine/metoprolol) in patients with Prader-Willi syndrome (PWS). PWS is a genetic disorder that begins in childhood. People with Prader-Willi syndrome always feel hungry, which can often lead to obesity and type 2…

A team of researchers was able to restore the normal level of genes that are usually silenced in Prader-Willi syndrome (PWS) in patient neurons grown in the lab. The study, led by researchers at the University of Connecticut, is titled “Zinc finger protein 274 regulates imprinted expression…