Gene therapy might soon offer a new option for children with a rare genetic disorder that damages tissues throughout the body, researchers are reporting.
Nov 18, 2021
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(HealthDay)—Nexviazyme (avalglucosidase alfa-ngpt) was approved for the treatment of the rare inherited disorder late-onset Pompe disease in patients 1 year of age and older, the U.S. Food and Drug Administration announced ...
Aug 11, 2021
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An international research group, led by Jamey Marth, Ph.D., a professor at Sanford Burnham Prebys, has shown that the Neuraminidase 3 (Neu3) enzyme is responsible for the onset and progression of colitis—a chronic digestive ...
Jul 16, 2021
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An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National ...
May 12, 2021
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Rare diseases are sometimes the most difficult to treat because of a lack of research and fewer participants to study.
Apr 16, 2021
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Researchers at the Indian Institute of Science (IISc) have developed artificial enzymes that can successfully block reactivation and replication of the Human Immunodeficiency Virus (HIV) in the host's immune cells.
Apr 1, 2021
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Results of a world-first Canadian pilot study on patients treated with gene therapy for Fabry disease show that the treatment is working and safe.
Feb 25, 2021
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A study aiming to develop a new therapeutic technique could bring a revolution in our approach to treating rare, fatal Sanfilippo syndrome, a disorder that affects children as young as 2 years old and leads to childhood dementia ...
Feb 24, 2021
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All the cells in the human body share the same genes. But how our genes are expressed determines whether a cell becomes a brain cell or a liver cell. In addition, changes in gene expression often play a significant role in ...
Jan 25, 2021
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Hop-based dietary supplements that many women use to ease the night sweats and hot flashes commonly reported during menopause aren't likely to cause drug interactions, new research from Oregon State University's Linus Pauling ...
May 19, 2020
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