With 250 rare diseases newly identified every year, scientists can barely keep up — even as the healthcare system fails millions of Americans whose rare diseases have already been diagnosed. That’s the warning from Christopher P. Austin, MD, director of the National Center for Advancing Translational Studies (NCATS) at…
Experimental immunotherapy laquinimod reduced brain atrophy in the caudate nucleus, one of the most affected brain areas in Huntington’s disease, and other cerebral regions but it did not improve motor function in recently diagnosed patients, LEGATO-HD study findings show. Results from the trial were recently discussed at the…
Mild genetic or environmental changes that upset the circadian clock (our internal sleep-wake cycle) may work to diminish the toxicity of mutant huntingtin, the protein involved in Huntington’s disease and nerve cell death, an early study in a fruit fly model found. The study, “Circadian Clocks Function in Concert…
A protein that can specifically bind to mutated forms of huntingtin and prevent its production — while preserving the normal versions of this protein — may represent a new strategy to attack Huntington’s disease. Researchers from Sangamo and Shire are going to present results from preclinical studies of…
Although many patients with Huntington’s disease (HD) experience pain, they report this problem has less of an effect on their daily lives than it does for the general population, a review study has found. Due to the areas of the brain affected in Huntington’s, patients…
DYST 201, a gene therapy targeting the mutated forms of the gene responsible for Huntington’s disease, has shown promising results, Dystrogen Therapeutics announced. The results will be presented at two meetings in Krakow, Poland — the July 6-11 44th FEBS Congress and the June 11-16 24th Annual Meeting of the RNA Society.
The U.S. Food and Drug Administration (FDA) has given fast track designation to AMT-130, uniQure’s gene therapy candidate for the treatment of Huntington’s disease. The designation helps to speed the development, testing, and review of therapies for serious diseases with high unmet medical need. “Achieving Fast Track Designation from the…
Aiming to raise awareness about Huntington’s disease (HD) while also commemorating a decade of community service, HD Reach presented its annual Education Day April 6 in Raleigh, North Carolina. Themed “Looking Back, Reaching Forward,” the event was held at the McKimmon Center at North Carolina State University. The non-profit…
Genistein, a potent stimulator of cells’ natural “cleaning system” was able to reduce the levels of mutant huntingtin protein in skin cells from Huntington’s disease patients, a study has found. The study, “Genistein induces degradation of mutant huntingtin in fibroblasts from Huntington’s disease patients,” was published in…
Combining stem cell and gene therapy can ease symptoms and lengthen survival time in a mouse model of Huntington’s disease, according to recent research. The study, “Combination of stem cell and gene therapy ameliorates symptoms in Huntington’s disease mice,” was published in the journal Regenerative Medicine. Huntington’s is caused…
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