News

At least 800 people representing some 45 countries are soon expected to gather in Austria’s capital city, Vienna, for ECRD 2018, the 9th European Conference on Rare Diseases and Orphan Products. The May 10-12 meeting is sponsored by Eurordis, the Paris-based group that defines itself as a “patient-driven alliance”…

Treatment with investigational medication VX15/2503 halts disease-related loss of brain volume and improves its metabolic activity in Huntington’s disease patients, according to a Phase 2 clinical study. The research, “Clinical Development of VX15/2503 Anti-Semaphorin 4D Antibody as a Potential Treatment for Huntington’s Disease,” was presented by Elizabeth Evans, PhD,…

The investigative therapy IONIS-HTTRx effectively reduced levels of the protein responsible for Huntington’s disease in early-stage patients, according to the findings of an ongoing Phase 1/2 clinical trial. The results also revealed that IONIS-HTTRx is generally safe and well-tolerated by patients, further demonstrating its potential as an alternative therapeutic option…

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,…