Mouse Models Prove Success of Gene Therapy for the Treatment of Huntington’s Disease

Research has recently been published in Nature Communications that shows the success of a new gene therapy in the treatment of Huntington's Disease. This therapy, an adeno-associated virus-based gene therapy, was tested…

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Neuroscientists Use Genetic Screen to Identify Huntington’s Disease Drug Targets
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Neuroscientists Use Genetic Screen to Identify Huntington’s Disease Drug Targets

By Jodee Redmond from In The Cloud Copy Massachusetts Institute of Technology (MIT) researchers have recently announced they have identified hundreds of genes that are required for neuron survival. The…

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A Scientist Dedicated Her Life to Researching Huntington’s Disease Before Facing a Diagnosis Herself
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A Scientist Dedicated Her Life to Researching Huntington’s Disease Before Facing a Diagnosis Herself

According to a story from Yahoo News, Dr. Nancy Wexler spent around 20 years in Venezuela conducting pivotal research on a deadly rare genetic disorder: Huntington's disease. On Lake Maracaibo,…

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Experimental Treatment for Huntington’s Disease Earns Orphan Drug Designation in the EU
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Experimental Treatment for Huntington’s Disease Earns Orphan Drug Designation in the EU

According to a story from globenewswire.com, the biotechnology company Emerald Health Pharmaceuticals Inc., has announced recently that its experimental drug EHP-102 has earned Orphan Drug designation from the European Medicines…

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Huntington’s Disease: Setting Expectations and Risks About Clinical Trials
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Huntington’s Disease: Setting Expectations and Risks About Clinical Trials

According to a story from EurekAlert! a recent study that was published in the Journal of Huntington's Disease has concluded that while this rare disease community is excited about what the latest…

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This Huntington’s Disease Trial Could be a Breakthrough–But Not Everyone That Needs Help Can Get In
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This Huntington’s Disease Trial Could be a Breakthrough–But Not Everyone That Needs Help Can Get In

According to a story from statnews.com, the family of 46 year old Tammy Stewart has been plagued by Huntington's disease, an ultimately fatal, progressive, degenerative disease that causes decades of…

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New Partnership Aims to Develop Treatment for Huntington’s Disease
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New Partnership Aims to Develop Treatment for Huntington’s Disease

According to a story from ncbiotech.org, a new partnership between Vertex Pharmaceuticals and RNA-based therapeutics company Ribometrix aims to develop potential treatments for serious diseases, including Huntington's disease, which is…

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New Study Finds Genetic Instability in Brain Cells as Key Trigger for Huntington’s Disease

According to a story from Medical Xpress, a recent study has determined that the onset of Huntington's disease symptoms is triggered by genetic instability in a specific sequence of DNA…

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DNA Sequencing Took Years, Now it Takes Hours; As a Result, Researchers Have Discovered The Cause of Four Rare Diseases

Researchers at the University of Tokyo have discovered that one segment of DNA is responsible for four rare diseases. As reported in Science Daily, the university team also believes that…

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A New Way to Study Disease: Scientists Have Recreated a Blood-Brain Barrier Outside the Body
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A New Way to Study Disease: Scientists Have Recreated a Blood-Brain Barrier Outside the Body

  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 FDA, Congress, A Young Woman Dying of ALS, Her Physician, and Her Parents Are All Struggling Over Access to an Untested Therapy

1032 words 6% vs 3711 words 2% The Hermstads of Spencer, Iowa lost Jaci’s twin to ALS and now they must watch Jaci, rapidly descend into the depths of the…

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CRISPR-Cas9: Scientists and Patients Have Philosophical Differences

  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…

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Study Suggests Brain Enzyme Might Form Tubes That Transport Huntington’s Disease Protein
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Study Suggests Brain Enzyme Might Form Tubes That Transport Huntington’s Disease Protein

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,…

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To Know or Not to Know: Your Prerogative as Someone at Risk for Huntington’s Disease

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…

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Findings From Latest Trial Could Herald Progress in Developing a Treatment for Huntington’s Disease
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Findings From Latest Trial Could Herald Progress in Developing a Treatment for Huntington’s Disease

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…

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Study Aims to Improve Patient Centricity in Huntington’s Disease Clinical Trials

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…

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