Columns

Neuron-targeted treatment based on zinc-finger DNA-binding proteins — called ZFPs — was found to lower brain levels of mutant huntingtin (mHTT) protein in a Huntington’s disease mouse model. The zinc-finger proteins rescued behavioral deficits and extended survival in these mice, the study demonstrated. ZFPs are among the most abundant…

For months, people from across the U.S. have been working hard with members of the Huntington’s Disease Society of America’s (HDSA) advocacy caucus to convince members of Congress to pass the Huntington’s Disease Parity Act. My wife, Jill, who is gene-positive…

The U.S. Food and Drug Administration (FDA) has agreed to review Neurocrine Biosciences’ supplemental new drug application (sNDA) for valbenazine to treat chorea caused by Huntington’s disease. “This sNDA filing advances our effort to bring a potential new treatment option to the many thousands of people experiencing chorea associated with…

Researchers have developed a new therapy that reduced the toxic buildup of the mutant huntingtin (HTT) protein — the hallmark of Huntington’s disease — in lab-grown neurons from Huntington’s patients and in a mouse model of the disease. The CRISPR-based therapy was designed to specifically target the HTT gene’s messenger…

The other day, my wife, Jill, shared some life advice to our 25-year-old nephew, Dylan, who’s living and working in a monastery in Eastern Europe, trying to figure out what he wants to do in his life. He’s got many gifts. He’s a talented artist and musician. He loves…

Branaplam (LMI070), an experimental oral therapy for Huntington’s disease that’s currently in early clinical trials, reduces levels of the mutant huntingtin protein by interfering with a process called mRNA splicing, a new study reveals. “Here, we delineated the mechanism of action of Branaplam,” researchers wrote. The study, “…

Recently, my wife, Jill, and I were talking about a book I was reading. She commented that I don’t just read books, I devour them. As the conversation continued, we started talking about how both of our fathers liked to read as well. Jill’s dad passed away in 2011…

As I write this first paragraph, I’m in the living room. My wife, Jill, is on the sofa next to me doing her nails, and my dog, Baby Girl, is several feet away on the floor under the piano, sleeping on her side. I can hear her breathing. By…

Deciding whether to undergo genetic testing for Huntington’s disease (HD) is a personal choice. As Ana Pena reports in a 2019 article for Huntington’s Disease News, researchers have found that “as many as 90% of individuals who have a parent with Huntington’s choose not to take a…

Recently, my wife, Jill, who is gene-positive for Huntington’s disease (HD), took a trip to Boston to participate in an observational HD study. She had been looking forward to it in part because she’d be able to visit her brother and his family and stay at our daughter‘s…