An abundance of events are afoot around the world to mark Rare Disease Day 2020 on Feb. 29. The activities are focused on heightening awareness about rare diseases and the hundreds of millions of individuals they are thought to affect. Patients, caregivers, and advocates worldwide will sport denim ribbons…
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Digital gait measurements collected through sensors can be used as a biomarker for walking impairments in people with Huntington’s disease (HD), a new study suggests. Individuals with HD were found to have smaller stride lengths and slower walking speeds, with more variation in their walking patterns, compared…
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Artificial Intelligence Can ‘See’ Progression of Ills Like Huntington’s in Blood Sample, Study Says
Artificial intelligence (AI) can predict disease progression using blood samples, a first long-term study of the molecular changes underlying neurodegeneration reports. Scientists at McGill University trained an AI algorithm to analyze blood and post-mortem brain tissue from 1,969 people with Alzheimer’s and Huntington’s disease. From the blood…
Neural stem cell researchers at the University of California, Irvine, (UCI) will collaborate with AgeX Therapeutics to develop cell-based therapies to treat Huntington’s disease and other neurological disorders. Leslie Thompson…
The U.S. Food and Drug Administration (FDA), a vast government bureaucracy, employs about 17,500 people and had a budget of $5.7 billion in 2019. Yet even with its enormous resources, the FDA these days relies more and more on patients to…
The gleaming new Dutch headquarters of the European Medicines Agency (EMA), fronting Domenico Scarlattilaan in Amsterdam’s suburban Zuidas business district, finally opened for business last month — just over two years after the European Union decided to relocate the EMA to the Netherlands in the wake of Brexit.
The European Medicines Agency (EMA) granted orphan designation to Emerald Health Pharmaceuticals’ (EHP) EHP-102 for the treatment of Huntington’s disease. Orphan status is given to medications designed to treat, prevent or diagnose rare life-threatening or chronically debilitating disorders that affect no more than five…
Neurons in the striatum — a brain region responsible for motor control — require a functional huntingtin (HTT) gene to survive and remain healthy during aging and to allow proper communication between nerve cells, according to a mouse study. The study, “Striatal Projection Neurons Require…
Rexulti (brexpiprazole), an antipsychotic medicine approved for schizophrenia and in some cases depression, eases motor and psychiatric symptoms of Huntington’s disease, according to a case study. The study, “Effectiveness of Brexpiprazole in the Treatment in a Patient with Huntington’s Disease,” was published in the…
AMT-130, uniQure’s experimental gene therapy candidate for Huntington’s disease, halts shrinkage of certain brain regions and helps preserve cognitive function in a mouse model of the disease, preclinical data show. Additionally, a single dose of AMT-130 reduced the levels of mutated huntingtin (HTT) — the protein that…
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