The European Medicines Agency (EMA) has granted orphan medicinal product designation to AMT-130, uniQure’s gene therapy candidate for the treatment of Huntington’s disease. AMT-130 is an experimental gene therapy that is being evaluated in studies designed to support an investigational new drug (IND) application with the U.S.
uniQure expects to advance the clinical development of its Huntington’s disease investigational gene therapy, called AMT-130, during 2018. The company says it plans to file an investigational new drug application (IND) with the U.S. Food and Drug Administration (FDA) in the second half of 2018. The first human clinical trial…
Keeping a strict eating schedule may help improve the quality of life of patients with Huntington’s disease and other neurodegenerative disorders, a mouse study suggests. The study, “Time restricted feeding improves circadian dysfunction as well as motor symptoms in the Q175 mouse model of Huntington’s disease,” appeared…
MMJ BioScience has filed a patent invention with the U.S. Patent and Trademark Office for its part-cannabinoid treatment for patients with cannabinoid-responsive diseases, including Huntington’s disease and multiple sclerosis. According to the announcement by MMJ BioScience — an affiliate of MMJ International Holdings, the patent will protect…
Huntington’s disease (HD) patients respond to endurance training much like anyone else, by increasing muscular energy metabolism, say Swiss researchers who suggest such training might help delay muscular dysfunction in patients. Their study, “Effects of endurance training on skeletal muscle mitochondrial function in Huntington disease patients,” appeared in the…
Researchers found biomarkers — molecules with elevated levels — in people who carry the Huntington’s disease (HD) gene who have not yet shown symptoms of the disease. These markers may allow for the testing of new treatments before the onset of Huntington’s in susceptible individuals. The study, “MicroRNAs in…
Researchers have found that bexarotene, an existing, approved drug used to treat cancer, may also be an effective therapy for Huntington’s disease. The study, “PPARδ activation by bexarotene promotes neuroprotection by restoring bioenergetic and quality control homeostasis,” appeared in the journal Science Translational Medicine. Researchers used…
People with Huntington’s disease in the U.K. are pushing for more open communication regarding assisted dying between clinicians, patients, and their families, a small research study indicates. A first-of-its-kind study in the United Kingdom, where assisted dying is illegal, looked into the attitudes of people with the debilitating disease, which…
Aberrant metabolism of urea is a potential first event promoting Huntington’s disease (HD), a study with a sheep model and human brain samples showed. The findings suggest that lowering brain levels of urea is a potential therapeutic avenue for HD. The report “Brain urea increase is an early Huntington’s…
The chemical compound manganese (Mn) has been reported to take part in the underlying mechanism of Alzheimer’s disease. However, little is known about its involvement in the development of other neurodegenerative diseases. In the study titled “Manganese in manganism, Parkinson’s disease, Huntington’s disease, amyotrophic lateral sclerosis, and…
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