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…
News
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…
Raman spectroscopy, an analysis technique that uses light, identified a specific molecular fingerprint in the blood of people with Huntington’s disease, a study has found. This technique may be useful as a non-invasive diagnostic tool to track disease progression and as…
Railway signalman Sandy Patience has witnessed the devastating effects of Huntington’s disease on family members. When he learned two years ago that he, too, inherited the neurodegenerative disorder, the news was crushing. But now, as one of the first Scotland residents to participate in an international Phase 3 trial…
As many as 80 experts from a dozen countries will gather next month in Vienna for the European Conference on Controversies in Huntington’s Disease (ECCH2020). The Feb. 13–14 meeting “aims to increase awareness and interests around the main aspects” of Huntington’s disease, which according to the European…
Efforts to understand brain development in young people at risk for Huntington’s disease have been given a boost by an $18 million grant awarded by the National Institute of Neurological Disorders and Stroke, a division of the…
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