First Two Patients Treated with AMT-130 for Huntington’s Disease Trial
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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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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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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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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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30 Million Euros Allocated to Fund Precision Medicine Research Projects for Neurodegenerative Diseases

Precision mNedicine is the latest craze in rare disease research. Thankfully for patients, it is completely, 100% centered on improving the quality of their care. Unlike most typical healthcare practices,…

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Is There a Genetic Condition in Your Family?
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Is There a Genetic Condition in Your Family?

Is there a genetic condition in your family? Are you concerned about the potential for passing this on to your children one day? Do you know if you are a…

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Five Benefits of the New Pig Models of Huntington’s Disease, According to Scientists
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Five Benefits of the New Pig Models of Huntington’s Disease, According to Scientists

New pig models of Huntington’s disease have been developed, and they are expected to have several benefits compared to existing rodent models of the condition. Read the source article here…

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A Drug That Can Mimic Exercise May Be Closer Than You Thought
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A Drug That Can Mimic Exercise May Be Closer Than You Thought

Scientists in California at Salk Institute for Biological Studies are currently developing a drug that can mimic the effects in the body usually obtained from exercise, reports the National Post.…

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