With early data on AMT-130 for Huntington’s disease reportedly looking promising, uniQure said it’s planning to meet early next year with regulatory authorities to discuss the late-stage development of its gene therapy candidate. By that time, more data on the ongoing Phase 1/2 clinical trials testing the therapy…
My wife, Jill, and I recently celebrated 15 years of marriage. When we said “I do” all those years ago, I couldn’t have guessed what would eventually happen to her: She is being robbed of her words. Or, as she puts it, words are “escaping” her. Jill will be…
Increased levels of the stress response protein MTF1 suppressed the toxic effects of the mutant huntingtin (mHTT) protein — the underlying cause of Huntington’s disease — in cellular and animal models of the neurodegenerative condition, a study showed. Specifically, MTF1 was found to counteract mHTT-associated oxidative stress, a type…
My wife, Jill, has always been stubborn, to put it lightly. She won’t admit that something is too hard for her. Ever. “No, I got it” is her favorite saying. But since she was diagnosed with Huntington’s disease in 2018, I haven’t been sure if stubbornness is…
People carrying fewer Huntington’s disease-causing CAG repeats in the HTT gene — who traditionally are thought to have milder disease — show similar cognitive deficits to those with more CAG repeats, a study found. However, patients with fewer repeats are less likely to show Huntington’s characteristic involuntary muscle contractions,…
The other day, a lightbulb went off when I thought about the things that Huntington’s disease might rob from me. I’m married to someone who was diagnosed with Huntington’s in 2018, and I often focus on what the disease will steal from her (or us). But I hadn’t…
When young, healthy progenitors of neuron-supporting cells called glia are transplanted into the adult brain, they can grow and replace diseased glial cells, including those carrying a Huntington’s disease-causing mutation, a study in mice reports. “These findings have strong therapeutic implications, as they suggest that in the adult human…
What do you say — in under a minute! — to someone about a rare disease? How do you convey the needs of people with Huntington’s disease (HD), knowing how terrible it is because you’ve spent years watching what it does to people you love? How can you explain…
Huntington’s disease could develop at earlier ages in people using calcium channel blockers to control hypertension, a study that looked into genes encoding targets of blood pressure-lowering medications reported. Findings may have important implications for managing high blood pressure in people at risk of Huntington’s, identified before disease…
I love the random questions my wife, Jill, asks me. One such query recently led to a deep and productive conversation. Jill asked me if I thought that anyone could become a good writer. I knew there was a story behind the question, so I asked if she’d share…
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