Study Findings Suggest that WAYLIVRA Could be an Effective Treatment for Familial Partial Lipodystrophy
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Study Findings Suggest that WAYLIVRA Could be an Effective Treatment for Familial Partial Lipodystrophy

According to a story from globenewswire.com, Akcea Therapeutics and Ionis Pharmaceuticals recently announced the release of top line results from a clinical study. This trial tested the drug WAYLIVRA as…

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Company Releases Data From Open Label Trial of Experimental Fragile X Syndrome Treatment
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Company Releases Data From Open Label Trial of Experimental Fragile X Syndrome Treatment

According to a story from globenewswire.com, the drug company Zynerba Pharmaceuticals, Inc. has announced that the data from its phase 2 open label trial has been officially published. This clinical…

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Duke Neurologist Receives Grant to Investigate Origins of Chronic Inflammatory Demyelinating Polyneuropathy
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Duke Neurologist Receives Grant to Investigate Origins of Chronic Inflammatory Demyelinating Polyneuropathy

According to a publication from Duke University, Dr. Karissa Gable, a neurologist at the University, has received a one-year research grant from the GBS|CIDP Foundation International to study the activity…

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Making History: First Patient in Connecticut Receives Spinal Muscular Atrophy Gene Therapy

According to a story from Connecticut Children's, the hospital is one of the very first in the country to administer Zolgensma, a recently approved gene therapy for the rare genetic…

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Study Findings Suggest Concentration of Immune Cells Could Help Predict Systemic Sclerosis
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Study Findings Suggest Concentration of Immune Cells Could Help Predict Systemic Sclerosis

According to a publication from Scleroderma News, a recent study suggests that blood concentrations of certain immune cells could correlate with the severity of cases of systemic sclerosis. About Systemic…

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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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FDA Approves an Experimental Treatment of Various Blood Malignancies for Clinical Trials

According to a story from First World Pharma, the biopharmaceutical company Mustang Bio, Inc. recently announced that the US Food and Drug Administration (FDA) has approved their investigational new drug…

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