The U.S. Food and Drug Administration (FDA) has granted Voyager Therapeutics permission to start a clinical trial testing VY-HTT01,…
Articles by Marisa Wexler, MS
A chaperone protein called DnaJB8 has an internal molecular “switch” that likely regulates how cells dispose of protein aggregates, like…
A new international consortium, Stem Cells for Huntington’s Disease (SC4HD), has been formed to help facilitate the development of stem…
Note: This story was updated Jan. 14, 2021, to note that pridopidine is no longer called Huntexil. Treatment with…
The National Organization for Rare Disorders (NORD)’s RareLaunch training program will host two days of…
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…