The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of…
Articles by Marisa Wexler, MS
The U.S. Food and Drug Administration (FDA) has given orphan drug designation to the oral therapy branaplam (LMI070) as…
A majority of rare disease patients using telehealth during the COVID-19 pandemic thought the experience positive, and many would like…
Vaccinex’s investigational antibody pepinemab showed good tolerability in a Phase 2 clinical trial of people with late prodromal (before…
People who will develop Huntington’s disease have subtle biological evidence of brain damage decades before the onset of symptoms,…
Among people with Huntington’s disease living in India, psychiatric problems are common but routine clinical follow-up is lacking, a study…
Austedo (deutetrabenazine) can be safely taken at higher than the maximum dose now approved to control involuntary movement in…
A novel class of proteins, dubbed heat-resistant obscure (Hero) proteins, can protect other proteins from a variety of cellular stresses,…
Multiple government agencies have cooperated to allow MMJ International Holdings to import cannabis extracts to the United States, for…
People with Huntington’s disease who develop psychosis tend to have worse cognitive and behavioral disturbances, reduced ability to…