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CHDI Foundation has teamed up with PTC Therapeutics to try to advance PTC’s work on small-molecule compounds that can reduce the production of huntingtin, the protein responsible for Huntington’s disease. The compounds have shown promise. They decreased the protein in a mouse model carrying the human huntingtin gene. And they…

A little-known government entity within the National Institutes of Health (NIH) is helping to lead U.S. efforts to speed up the development of therapies for some 7,000 rare diseases. The Office of Rare Diseases Research (ORDR), headquartered in Bethesda, Maryland, was established in 1993 within the NIH Office of the…

Mutations linked to Huntington’s disease cause more extensive remodeling of functional connectivity in various regions of the brain than previously thought, affecting patients’ motor and cognitive skills, research looking at whole brain connectivity in disease carriers reports. These findings — in “Whole-Brain Connectivity in a Large Study of Huntington’s…

Potential immunotherapies are showing some positive early results in clinical and preclinical tests, and may offer hope to treat Huntington’s disease, according to a recent review published in the journal Molecular Psychiatry. The review is titled “Are immunotherapies for Huntington’s disease a realistic option?” Huntington’s is…

Scientists recently uncovered the reason why patients with Huntington’s disease have up to 80 percent less cancer than the general population, a discovery they now hope will lead to a new approach for cancer treatment. Their research found that the mutated huntingtin (HTT) gene in Huntington’s patients generates a class…

Blocking an enzyme called PIP4Ky has the ability to reduce levels of mutant huntingtin protein and clear toxic aggregates of the protein, suggesting a potentially effective therapeutic strategy against Huntington’s disease, according to a study. The study, “Inhibition of PIP4Kγ ameliorates the pathological effects of mutant huntingtin…

Cannabinoids can help to treat motor symptoms like dystonia in patients with early-onset Huntington’s disease, a small case study reports. The study, “Cannabinoids for Treatment of Dystonia in Huntington’s Disease,” appeared in the Journal of Huntington’s Disease. Huntington’s disease is characterized by movement disorders, cognitive deterioration,…

Three organizations fighting neurodegenerative diseases have teamed up to challenge researchers around the world to come up with new treatment approaches for central nervous system (CNS) disorders. The Huntington’s Disease Society of America (HDSA), the ALS Association, and Teva Pharmaceuticals have launched the crowdsourcing effort, called the …

A modified version of the genome-editing CRISPR-Cas9 system may prevent the production of faulty huntingtin protein, moving closer to treatment for Huntington’s disease. The study, “Precise Excision of the CAG Tract from the Huntingtin Gene by Cas9 Nickases,” was published at Frontiers in Neuroscience. The new…

Genetic sequencing and the speed with which it can help diagnose a child’s disease — in addition to revealing the genes that cause at least half of the 7,000 rare diseases currently known — was the focus of a discussion by three top New York geneticists. The Feb. 28 conference,…