News

European Initiative Targets Diagnosis, Treatment of Rare Diseases

A new international consortium based in Paris, and funded largely by the 28-member European Union, intends to speed the diagnosis of rare diseases, while also accelerating the development of treatments for the 95% of such illnesses that currently don’t have one. The European Joint Programme on Rare Diseases (EJP…

Investigational Therapy May Alleviate Sleep Disorder, Cognitive Decline in Children with PWS

Pitolisant, an investigational anti-sleepiness therapy in the U.S., may help reduce daytime sleepiness and improve cognitive function in children with Prader-Willi syndrome (PWS), a case series suggests. The study, “Cognitive Improvements in Children with Prader-Willi Syndrome Following Pitolisant Treatment — Patient Reports,” was published in the Journal of…

NORD Honors Industry, Patient Advocates at Rare Impact Awards Gala

A violinist with vasculitis, two Texas politicians and a pharmaceutical company whose marijuana-derived therapy helps kids with Dravet syndrome were among winners of the 2019 Rare Impact Awards. Officials of the National Organization for Rare Disorders (NORD) presented the awards during a June 22 dinner attended by…