The U.S. Food and Drug Administration has approved Takeda Pharmaceuticals Adzynma, the first recombinant protein product indicated for prophylactic or on-demand enzyme replacement therapy (ERT) in adult and pediatric patients ...
Nov 14, 2023
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The UK regulatory authorities have approved the first ever trial of a revolutionary gene therapy for young children diagnosed with Hunter syndrome, a devastating rare lysosomal storage disorder.
Oct 30, 2023
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Using a protocol developed at UC San Francisco, physicians have successfully treated a fetus with a devastating genetic disorder for the first time, and the child is now thriving as a toddler, a case study in the New England ...
Nov 10, 2022
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The advent of small-molecule targeted therapies, a decade ago, revolutionized the treatment of metastatic melanoma, provided that the tumors carry the mutations to respond to these treatments. However, despite a remarkable ...
Sep 22, 2022
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The U.S. Food and Drug Administration has approved Xenpozyme (olipudase alfa) for pediatric and adult patients with acid sphingomyelinase deficiency (ASMD), a rare genetic disease.
Sep 5, 2022
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Fluoxetine, a common antidepressant, inhibits the SARS-CoV-2 coronavirus in cell cultures and in preparations from human lung tissue. This was demonstrated by researchers at Julius-Maximilians-Universität (JMU) Würzburg ...
Aug 17, 2022
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The transgender population encounters numerous barriers to receiving proper medical care. Even when treatment is accessible, healthcare providers may lack comprehensive knowledge on how to treat them either due to insufficient ...
Apr 26, 2022
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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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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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