Finding treatments and potential cures for rare diseases is crucial, but so is the quality of patients’ lives — a…
Articles by Larry Luxner
The National Organization for Rare Disorders (NORD) will celebrate the 35th anniversary of both the 1983 Orphan Drug Act and…
The European Union isn’t doing enough to protect the 30 million or so people with rare diseases who live in…
At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for…
An advisory committee of the U.S. Food and Drug Administration has recommended approval of Epidiolex, an oral cannabidiol medicine,…
A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed…
Raising a child with Dravet syndrome is hard enough without the added stress of fighting with insurance companies to cover…
Genetic sequencing and the speed with which it can help diagnose a child’s disease — in addition to revealing the genes…
When it comes to rare diseases, one that definitely makes the list is spinal muscular atrophy with respiratory distress —…
In his 10 months on the job, Commissioner Scott Gottlieb of the U.S. Food and Drug Administration is earning…