Researchers at Tel Aviv University have developed a new approach for using locked nucleic acids (LNAs)—a particularly stable type of RNA—to treat inflammatory bowel diseases such as Crohn's disease ...

On Thursday, the U.S. Food and Drug Administration (FDA) approved Ionis Pharmaceuticals, Inc.’s (NASDAQ:IONS) Dawnzera (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) ...

Dawnzera (donidalorsen) is a new medicine approved to help prevent attacks of a rare genetic swelling disorder known as hereditary angioedema (HAE) in people aged 12 or older. Affecting about 7,000 ...

Children who develop deep wrinkles, stunted growth, and rapidly aging bones and blood vessels as early as 1 to 2 years of age may be suffering from Hutchinson-Gilford Progeria Syndrome (HGPS), a rare ...

In recent years, the scientific community has made significant strides in the field of gene editing, particularly through the development of the CRISPR (Clustered Regularly Interspaced Short ...

Parkinson’s disease is associated with the buildup of a particular brain protein that currently has no approved therapies addressing it. Novartis was among the companies pursuing this target, but its ...

The vector consists of a polyethylene glycolylated liposomal outer coat (providing immune evasion and prolonged circulation), a spindle-shaped nanomotor (resembling a flagellum, facilitating targeted ...

The United States Food and Drug Administration has just approved the first-ever clinical trial that uses CRISPR-Cas13 RNA editing. Its aim is to treat an eye disease called wet age-related macular ...

Children who develop deep wrinkles, stunted growth, and rapidly aging bones and blood vessels as early as 1 to 2 years of age may be suffering from Hutchinson-Gilford Progeria Syndrome (HGPS), a rare ...

On Thursday, the U.S. Food and Drug Administration (FDA) approved Ionis Pharmaceuticals, Inc.’s (NASDAQ:IONS) Dawnzera (donidalorsen) for prophylaxis to prevent attacks of hereditary angioedema (HAE) ...