Amicus Therapeutics and the FDA Have Met to Discuss the Path for a Potential Pompe Disease Therapy
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Amicus Therapeutics and the FDA Have Met to Discuss the Path for a Potential Pompe Disease Therapy

  Amicus Therapeutics has met with the United States Food and Drug Administration to discuss the investigational drug AT-GAA, which is being researched as a potential treatment for Pompe disease.…

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A Scottish Political Party Has Passed a Motion About Myalgic Encephalomyelitis
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A Scottish Political Party Has Passed a Motion About Myalgic Encephalomyelitis

  The political party the Scottish Liberal Democrats has voted in support of a motion about myalgic encephalomyelitis (ME). This motion calls for several changes, including stopping graded exercise therapy…

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A Gene Therapy for Wet Age-Related Macular Degeneration has Progressed to Clinical Trials

The United States Food and Drug Administration has awarded Fast Track Designation to an experimental gene therapy for the treatment of wet age-related macular degeneration. For more detailed information, you…

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Researchers Are Trying to Understand the Link Between Specific Genetic Variants and Symptoms in Neurofibromatosis Type 1
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Researchers Are Trying to Understand the Link Between Specific Genetic Variants and Symptoms in Neurofibromatosis Type 1

An international study led by researchers at the University of Alabama Birmingham has investigated the link between a specific genetic mutation underlying neurofibromatosis type 1, and the symptoms patients with…

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