News

AMT-130 is first Huntington’s therapy to receive RMAT status

The U.S. Food and Drug Administration (FDA) has granted regenerative medicine advanced therapy (RMAT) status to AMT-130, an experimental gene therapy for Huntington’s disease, the first time a Huntington’s treatment candidate has received such a designation. The RMAT designation is given to therapies that seek to treat, modify,…

HDSA 2024: Study cites need for family psychological support

There’s a sore need to provide more psychological support to people living with Huntington’s disease and their families, not only around the time of testing and potential diagnosis, but throughout the different stages of the disease, according to a study released at the 2024 Huntington’s Disease Society…

HDSA 2024: CEO shares optimism for Huntington’s community

The outlook for the Huntington’s disease community has never been brighter, with better care options and promising new research already in progress, the president and CEO of the Huntington’s Disease Society of America said at the organization’s 39th annual convention, taking place in Spokane, Washington. “There has never…

FDA approves more Austedo XR one-pill-a-day tablets

The U.S. Food and Drug Administration (FDA) has authorized new dosage strengths of Austedo XR (deutetrabenazine) extended-release tablets for managing chorea, or involuntary movements, in adults with Huntington’s disease. Teva Pharmaceuticals’ Austedo XR is an extended-release formulation of Austedo (also by Teva) that is meant to…

Model may help develop medicines that cross blood-brain barrier

Researchers in the U.S. have created the first brain-like organoids, or mini-brains, that incorporate a working blood-brain barrier (BBB), which is the membrane that regulates which circulating substances can reach the brain and spinal cord, and that typically challenges the entry of medications. Called a BBB assembloid, the human model…

New viral carrier shows promise for brain-targeted gene therapies

Scientists at the Broad Institute of MIT and Harvard have engineered a harmless adeno-associated virus (AAV) that can efficiently reach the brain, potentially improving the efficacy of brain-targeted gene therapies for neurological conditions such as Huntington’s disease. Current AAVs that deliver gene therapies to cells in the body via…