First Two Patients Treated with AMT-130 for Huntington’s Disease Trial

uniQure has recently began their Phase I/II clinical trial of AMT-130, a gene therapy for the treatment of Huntington's disease. Two patients are included so far, with one being given…

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It Just Makes “Antisense:” Using Genetic Medicine to Treat Rare Diseases
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It Just Makes “Antisense:” Using Genetic Medicine to Treat Rare Diseases

  As previously published in Scientific American, slightly after her first birthday, Emma Larson lost the use of her legs and started experiencing difficulty crawling. She was diagnosed with spinal muscular…

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Will Gene Therapy Finally Provide a Cure for Huntington’s Disease?

According to a recent article published in Pharmaceutical-Technology, uniQure’s gene therapy AMT-130 may lower levels of the huntingtin protein that causes the disorder. An abnormal gene is responsible for damage…

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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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Using PacBio SMRT Sequencing in Myotonic Dystrophy Type 1 and Other Rare Disorders

  Stéphanie Tomé is an investigator at the Sorbonne Université in Paris, France. A recent article in PacBio describes Tomé’s research into a disease that becomes progressively worse with each generation.…

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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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Researchers Might Have Just Made a Breakthrough Discovery for Treating Rare Proteinopathies

It's common knowledge among researchers who study diseases called toxic proteinopathies that this group of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…

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Researchers are Working to Repair a Small Step In a Pathway Involved in Neurodegenerative Diseases

It's common knowledge among researchers who study diseases called toxic proteinopathies, that this grou of disorders are the result of misfolded proteins that reside in cells. Certain proteins fail to…

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