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The blood-brain barrier is a “gatekeeper” that prevents toxins and foreign matter in the bloodstream from traveling to the brain. Check Orphan recently carried an article that had been…
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The Hermstads of Spencer, Iowa lost Jaci’s twin to ALS and now they must watch Jaci, rapidly descend into the depths of the same disease. Jaci has a rare…
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Calls to Ban CRISPR A scientist named He Jiankui recently shocked the scientific world with his announcement that he had created twin “CRISPR babies”. The babies, a result of…
According to a publication from Laboratory Equipment, a new animal study of mice with simulated Huntington's disease suggests that a protein associated with the symptoms of Huntington's can form small,…
Huntington's Disease Huntington's disease (HD) is a rare neurodegenerative disease. It progressively affects the patient's mobility and cognitive function. Unfortunately, a cure has yet to be discovered and the condition…
According to a story from Biopharma Dive, an investigational therapy for Huntington's disease that is in development could be the first step in the implementation of the first-ever disease modifying…
As the medical field continues to advance, treatments are becoming less and less "traditional" because researchers are uncovering unique ways to combat illnesses. Therapies range from pills, to surgery, to…
Neurodegenerative Diseases There are a wide range of neurodegenerative diseases including Huntington's disease and Parkinson's disease. These conditions typically affect the older population. As the world's population ages, more people…
A recent article in globenewswire.com news reported that the FDA has approved Fast Track Designation for AMT 130, an experimental gene therapy for the treatment of Huntington’s disease. The…
Huntington's Disease & SCAs Huntington's disease (HD) and spinocerebellar ataxia (SCA) are both neurodegenerative diseases caused by the same type of error in the body's DNA. Essentially, the CAG or…
A recent study published in Cell has exhibited the scientific community's negligence for conducting research that includes an adequate representation of non-Europeans. Specifically, it showed that 78% of patients in genomic…
Each year, Rare Disease Day (February 28th) incites individuals and organizations to investigate and reform their rare disease efforts. This year, the EU Commission has made drastic changes which should…
My name is Casey. I’m 27 years old and have a 50% chance of becoming symptomatic for a neurodegenerative disease in my 30’s (if my onset aligns with that of…
According to a story from Parkinson's News Today, a recent study describes the ability of an innovative smartphone app that uses touch-based input in order to detect the early signs…
Ionis Pharmaceuticals and Roche have been collaborating on a drug for Huntington's Disease which will treat the root cause of the condition. Currently, there are no such approved treatments, meaning…
University of Copenhagen researchers have been studying the cause of Huntington’s disease in order to potentially develop a treatment. They have determined that failure of glial cell maturation is a…
According to a story from ksl.com, a recent poll found that the majority of Americans are in support of using gene editing technology in order to protect newborns from genetic…
A diagnosis means everything for a rare disease patient. However, to process 200 patient records manually, it would take the most dedicated physician approximately 40 hours. That's one full work…
According to a story from news-medical.net, a team of researchers has found that patients who are more intellectually active can slow the neurodegeneration that appears in Huntington's disease. Such as…
Neurodegenerative disorders affect millions of people across the world. They include Amyotrophic Lateral Sclerosis, Parkinson's disease, Huntington's disease, and Alzheimer's disease, among others. For most neurodegenerative diagnoses, there are no effective treatments, no…
According to an article published on BioPortfolio, earlier this week, biotechnology company Vybion Incorporated secured orphan drug designation for its highly experimental Huntington's disease drug INT41. About Huntington's Disease Huntington's…
Huntington's Disease is a neurodegenerative condition caused by a genetic mutation. It results in progressive decline of motor skills. It also affects cognitive function. Patients experience decline for 15-20 years…
According to a story from Science Alert, an ugly and unpleasant tale involving the devastating rare genetic illness known as Huntington's disease will culminate in a lawsuit. The outcome of…
According to a story from abc.net.au, Cyril Wise watched in horror as his wife Kaye died of Huntington's disease in 2014. However, before his wife's death, Cyril was struck with…
The companies Genentech and Roche are working together to carry out a program of studies involving patients with Huntington’s disease (the huntingtin-lowering trial ASO program). One is a natural history…
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