Neurologists in the U.S. are optimistic about the potential for a new treatment for Huntington’s disease, but emphasize the need for therapies that halt nerve degeneration before symptom onset, according to a report by Spherix Global Insights. Most of the specialist physicians surveyed hoped for a gene therapy…
Forms of mutant huntingtin (mHTT) protein that don’t clump initially drive early disease progression in people with Huntington’s disease, according to a study wherein patient-derived cells were transplanted into newborn mice. These soluble forms of mHTT were found to cause structural changes in the neurons producing them and to…
People with Huntington’s disease were found to have a widespread deficiency of selenium — a trace mineral found naturally in foods such as seafood and Brazil nuts — in their brains, revealing a new potential therapeutic target, according to researchers. Alterations in the levels of other biologically active…
The U.S. Food and Drug Administration (FDA) will review Neurocrine Biosciences’ application of a new oral granule formulation of Ingrezza (valbenazine) for involuntary muscle contractions, or chorea, in adults with Huntington’s disease. The therapy was recently approved in the U.S. for Huntington’s-associated chorea in the form…
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…
The success of CDNF — fully, cerebral dopamine neurotrophic factor — in improving motor coordination in a mouse model of Huntington’s disease supports this protein’s development as a potential therapy for the rare disorder, according to a new study. In the study, CDNF was injected into the brain of…
Higher levels of healthy and mutant huntingtin protein in saliva, but not in blood, are significantly associated with worse motor symptoms of Huntington’s disease, a study showed. According to the investigators, these findings support the use of noninvasive saliva tests to monitor Huntington’s progression and predict clinical outcomes —…
The European Medicines Agency (EMA) has granted orphan drug status to SAGE-718 as a potential therapy for Huntington’s disease. The experimental therapy is being developed by Sage Therapeutics for treating cognitive impairment associated with Huntington’s and other neurological disorders. Orphan drug status is given to treatments with the potential to improve…
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…
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…
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