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…
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, “…
Abnormal red blood cells, known as acanthocytosis, were identified in four unrelated Chinese adults with Huntington’s disease — undermining researchers’ attempts to differentiate the neurodegenerative disorder from another similar genetic disease. Researchers had expected that the presence or absence of such abnormally shaped red blood cells would help…
Long-term treatment with Austedo (deutetrabenazine) safely led to sustained reductions in chorea for people with Huntington’s disease, including those who had switched from treatment with Xenazine (tetrabenazine). That’s according to about three years of follow-up data in the Phase 3 ARC-HD trial (NCT01897896), a study jointly conducted…
A data safety monitoring committee has recommended that enrollment of the highest dosing group resume in the European Phase 1/2 trial of AMT-130 in people with Huntington’s disease, but with additional safety measures in place. UniQure, the investigational treatment’s developer, announced in August that enrollment in…
A greater number of CAG repeats within the HTT gene — the genetic cause of Huntington’s disease — is linked to earlier disease onset and death, but is not an independent predictor of mortality in people with the neurodegenerative disease, a study shows. These findings suggest that “non-genetic factors…
The Hereditary Disease Foundation (HDF) is recognizing two scientists, Steven Finkbeiner and Natalia Barbosa, for their leadership in Huntington’s disease research. Both will speak at the organization’s 2022 Virtual Gala, set for Nov. 10 starting at 7 p.m. ET. The free, hourlong event will cover…
Palliative care training for all clinicians on an interdisciplinary team can increase the number of discussions about advanced care planning for people with Huntington’s disease, a new study shows. “To our knowledge, this is the first description of primary palliative care for Huntington’s disease in which all interdisciplinary team…
The activity of focal adhesion kinase (FAK), an enzyme known to play an essential role in nerve cell function, is low in the brains of people with Huntington’s disease, researchers discovered. Cell and mouse models revealed that the presence of abnormal huntingtin protein, the underlying cause of Huntington’s, blocked…
A single injection of Wave Life Science‘s experimental therapy WVE-003 was found, at either of the two lowest tested doses, to lead to reductions in the mutant huntingtin (mHTT) protein in people with Huntington’s disease — while leaving the healthy version of the protein unaffected. That’s according to…
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