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In rare cases, Huntington’s disease can develop spontaneously. In this case, the gene becomes altered by chance and is not inherited from either parent. This is known as a de novo gene ...

As hundreds of researchers, patient advocates and policymakers gather in Washington, D.C., this week for an advocacy fly-in on rare diseases, a notable gap has appeared on their schedule: an annual ...

Huntington’s disease was long thought to be caused by the slow buildup of a toxic protein, but new research has reveale | This week on "The Top Line," we hear from Steven McCarroll, Ph.D., whose ...

There is the disease itself: Huntington's is a rare genetic disease that causes the brain to break down over time. The most characteristic symptoms are involuntary movements of the face and limbs ...

Huntington’s disease is a brain disorder that can affect various functions, including the ability to swallow — particularly as the disease progresses. Huntington’s disease is a ...

Rare diseases (RD) are more common than you might think. For every 20 people you know, one is likely affected by a rare disease. Of the 7,000 known RD conditions, 80% have a genetic origin, yet over ...

ElevateBio is seeking out partnerships for its lead gene therapy after the construct reduced the levels of a toxic protein in ...

LETI-101 has demonstrated therapeutically relevant reductions of mutant huntingtin (HTT) protein, exceeding 80%, while preserving essential ...

Huntington’s disease is an inherited neurological disorder causing movement, cognitive, and behavioral issues. Effective management includes medical treatments, therapies, nutritional support ...