The U.S. Food and Drug Administration said on Tuesday it has approved SpringWorks Therapeutics' drug to treat a type of rare ...
Jones Trading initiated coverage on ProQR Therapeutics N.V. (NASDAQ:PRQR) with a Buy rating and set a price target of $11.00, representing significant upside from the current price of $2.19. The ...
The Food and Drug Administration (FDA) approved a new drug to treat a disorder causing the growth of non-cancerous tumors on ...
One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...
Researchers at Washington University School of Medicine in St. Louis have conducted a longitudinal study on an individual ...
A new UCLA Health study has discovered in mouse models that genes associated with repairing mismatched DNA are critical in eliciting damages to neurons that are most vulnerable in Huntington's disease ...
A new study has discovered in mouse models that genes associated with repairing mismatched DNA are critical in eliciting damages to neurons that are most vulnerable in Huntington's disease and ...
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One shot to lower cholesterol for life? Scientists unlock a groundbreaking gene therapyResearchers developed an epigenetic editing approach to silence the PCSK9 gene, achieving long-term cholesterol reduction ...
Background SLE is a systemic autoimmune disease with a large number of common risk gene variants, but several rare gene ...
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