Columns

SAGE-718 named orphan drug in EU for Huntington’s disease

The European Medicines Agency (EMA) has granted orphan drug status to SAGE-718 as a potential therapy for Huntington’s disease. The experimental therapy is being developed by Sage Therapeutics for treating cognitive impairment associated with Huntington’s and other neurological disorders. Orphan drug status is given to treatments with the potential to improve…

FDA approves once-daily formulation of Austedo for Huntington’s chorea

Austedo XR, a new extended-release formulation of Austedo (deutetrabenazine) has been approved by the U.S. Food and Drug Administration (FDA) to treat adults with chorea — involuntary muscle contractions — associated with Huntington’s disease. Austedo XR contains the same active agent as the original therapy, but is taken just once a day, with…

HDSA awards over $2M in grants to Center of Excellence network

The Huntington’s Disease Society of America (HDSA) is awarding 56 grants totaling more than $2 million to expand its Centers of Excellence network, which is dedicated to providing comprehensive care to people with Huntington’s disease. Dartmouth–Hitchcock Medical Center in New Hampshire joins last year’s total of 55 grant-funded Centers of Excellence. The…

Another punny Valentine’s Day for my honey

My objectives in this column are to acknowledge two upcoming holidays: Valentine’s Day, which my wife, Jill, doesn’t believe in (she thinks it’s manufactured and that love should be celebrated every day), and Rare Disease Day, on Feb. 28, which raises awareness and prompts change for…

Spark, Neurochase Collaborate on Brain Delivery Technology

Spark Therapeutics and Neurochase have joined forces to develop Neurochase’s proprietary delivery technology for gene therapies that target neurodegenerative diseases, such as Huntington’s disease. The technology, called Convection Enhanced Delivery (CED), allows for a controlled delivery of gene and other therapies directly into specific brain regions with the…