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…
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…
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…
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…
Lucy is now ten years old and stable, living her life as close to a normal ten-year old as possible. However, she was born with a super rare disease…
According to a story from fda.gov, the US Food and Drug Administration (FDA) has approved the drug pexidartinib (marketed as Turalio) as a treatment for tenosynovial giant cell tumor (TGCT)…
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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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…
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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