Autoimmune conditions like Type 1 diabetes, multiple sclerosis and rheumatoid arthritis arise when an immune system mistakenly attacks its own body's proteins, cells and organs. Not only do these conditions make the body ...
Nov 29, 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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A promising new therapy has – for the first time – reduced damage to the brain that can be caused by Sanfilippo B (MPS IIIB), a rare and devastating genetic disease, Los Angeles Biomedical Research Institute (LA BioMed) ...
Feb 12, 2014
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REGENXBIO Inc. today announced that gene transfer mediated by REGENXBIO's NAV AAV8 vectors resulted in sustained serum α-L-iduronidase (IDUA) expression, as well as correction of systemic features of MPS I, or Hurler syndrome, ...
Sep 30, 2014
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Among previously untreated adults with Gaucher disease type 1, a genetic disease in which there is improper metabolism due to a defect in an enzyme, treatment with the drug eliglustat resulted in significant improvements ...
Feb 17, 2015
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Two weeks and several political disasters ago, the House of Representatives passed the American Health Care Act of 2017, and soon lists of "pre-existing conditions" festooned news feeds. We all ticked off a few. But the lists, ...
May 22, 2017
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A new therapy involving stem cells may offer a better quality of life for patients with Hurler syndrome, a rare genetic disease that can cause crippling physical conditions and lead to early death.
Nov 16, 2017
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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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