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This week marks the launch of the “7,000 Mile Rare Movement,” a nationwide effort to raise money for research into the 7,000 known rare diseases that afflict at least 30 million Americans. The campaign kicks off Feb. 1 and culminates with Rare Disease Day on Feb. 28. Organized by…

Neurofeedback training may increase brain connectivity in patients with Huntington’s disease, possibly improving behavior and movement abnormalities, say British researchers at University College London. Their study, “Stimulating neural plasticity with real-time fMRI neurofeedback in Huntington’s disease: A proof of concept study,” appeared in the journal Human Brain Mapping.

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…