Small molecules developed by Design Therapeutics were able to reduce the activity of the disease-causing mutant gene in preclinical models of Huntington’s disease, according to an announcement from the company. “We are highlighting an exciting new program for Huntington’s Disease, an indication of tremendous unmet medical need,”…
I’ve come to a realization that has impacted my viewpoint about the importance of voting and advocating for political candidates who prioritize healthcare. It was prompted by my wife Jill’s diagnosis of Huntington’s disease (HD) and the challenges we’ve faced navigating the healthcare system as we deal with…
“The waste of money cures itself, for soon there is no more waste.” – M.W. Harrison The cognitive damage caused by Huntington’s disease has strongly affected my brother, Gavin. It’s part of the reason he is unable to manage his financial affairs on his own. We saw…
Prilenia Therapeutics plans to submit a marketing authorization application (MAA) by mid-year asking for approval in the European Union of pridopidine, an oral small molecule for treating Huntington’s disease. The planned filing with the Committee for Medicinal Products for Human Use, part of the European Medicines Agency (EMA),…
Recently, I’ve found myself grappling with a complex mix of emotions. On the one hand, I’m filled with admiration for the incredible strength and positivity that my wife, Jill, exudes every day in the face of Huntington’s disease (HD). On the other hand, there’s a lingering sadness…
“In sickness and in health, for as long as we both shall live.” These words took on deeper meaning for our family recently after I underwent genetic testing for Huntington’s disease. Huntington’s disease took our dad’s life in May 2020, and my brother, Gavin, was…
Two types of orally available small molecules ameliorated symptoms and slowed progression of Huntington’s disease in a mouse model, a study reported. The treatments effectively reduced the levels of the mutant huntingtin protein in the brain without affecting normal huntingtin. The mutant version causes Huntington’s, a neurodegenerative condition.
As regular readers of this column know, I love my wife, Jill, with all my heart. I love her simplicity. I love her sense of humor. And of course, I love that she’s the mother of our wonderful daughter, Alexus. Still, I have to admit…
“It’s your road, and yours alone. Others may walk it with you, but no one can walk it for you.” — Rumi, scholar and poet Huntington’s disease has so far affected four generations of our family. We lost our dad to Huntington’s in May 2020 and…
A first clinical trial of SKY-0515, an experimental treatment for Huntington’s disease, is starting to give multiple ascending doses to healthy adults randomized to the therapy rather than a placebo, Skyhawk Therapeutics, its developer, announced. Initiation of this part of the Phase 1 trial (ACTRN12623001161617), taking place in…
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