In a large Venezuelan family with Huntington’s disease, members with a rare variant in the WDFY3 gene developed symptoms up to 23 years later than expected, a study showed. The WDFY3 gene encodes Alfy, a protein that helps clear protein clumps, such as those driving Huntington’s and other proteinopathies…
Revir Therapeutics has been awarded a $4.6 million grant from the California Institute for Regenerative Medicine (CIRM) to support the development of a potential disease-modifying oral treatment for Huntington’s disease. The company also said it “reached a key program milestone,” identifying “a lead compound that meets key development…
Complex fatty molecules called gangliosides are key to the formation of cellular shipping containers that help brain nerve cells get rid of improperly folded proteins, including the mutated huntingtin (mHTT) protein that drives Huntington’s disease, a study shows. These containers are known as extracellular vesicles (EVs). “Now we know…
The U.S. Food and Drug Administration (FDA) no longer considers available clinical data sufficient to move forward with an application seeking approval of uniQure’s AMT-130 for Huntington’s disease, the company said. The company had planned to submit a biologics license application (BLA) early next year, based on…
Researchers in China have developed a new system to grow the specific type of brain cells that are lost in Huntington’s disease, and have seen benefits in testing it in a mouse model of the neurodegenerative disorder. Transplanting these cells into the brains of the animal model lessened the…
Three years of treatment with Ingrezza (valbenazine) led to rapid and sustained reductions in the severity of uncontrolled movements known as chorea for people with Huntington’s disease. That’s according to data from a long-term Phase 3 clinical trial called KINECT-HD2 (NCT04400331). Participants completing the three years of…
Up to two years of pridopidine treatment continues to slow Huntington’s disease progression among patients not taking certain standard treatments called antidopaminergic medications (ADMs). That’s according to a new analysis comparing long-term outcomes from pridopidine-treated people with Huntington’s in the Phase 3 PROOF-HD clinical trial (NCT04556656) against…
Available evidence does not indicate that treatments approved for people with Alzheimer’s disease can improve cognitive function in people with Huntington’s disease, according to a review study. “The existing literature does not provide sufficient evidence to support the use of cholinesterase inhibitors or memantine for managing cognitive symptoms in…
The Hereditary Disease Foundation (HDF), committed to advancing research on Huntington’s disease (HD) and potential treatments, has changed its name to Huntington’s Disease Foundation. “This name change is more than symbolic — it is a powerful reaffirmation of our purpose,” Meghan Donaldson, Huntington’s Disease Foundation CEO, said in…
uniQure’s experimental gene therapy AMT-130 safely and effectively slows the progression of Huntington’s disease, according to top-line, three-year data from two Phase 1/2 clinical trials. At 36 months, the use of high-dose AMT-130 was shown to slow Huntington’s progression by 75% — results the developer called “statistically significant”…
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