NOVATO, Calif. - Ultragenyx Pharmaceutical Inc. (NASDAQ: NASDAQ:RARE), a $4.08 billion market cap biotechnology company with 27% revenue growth in the past year, today unveiled new data indicating ...

Qure is planning soon to start enrolling patients in a second dose group as part of a clinical trial testing AMT-162 for SOD1 ...

Exagamglogene autotemcel (exa-cel) gene therapy offers a potential cure for severe sickle cell disease, now available on NHS.

Several companies—including JCR Pharmaceuticals, Denali Therapeutics and Regenxbio— have products in the pipeline that could ...

Featured data showcase the transformative potential of HSC gene therapy to enable cross-correction and restore enzymatic ...

Mucopolysaccharidosis Type II (MPS II) also known as Hunter Syndrome is when the body lacks the enzyme to break down sugar ...

The TP53 gene is a gene that is mutated in many cancers. It is the most common gene mutation found in cancer cells. A tumor-suppressor gene, TP53 codes for a protein that inhibits the development and ...

Some specialize in working with infants and babies, or with seniors. The respiratory therapy you need depends largely on whether you’re in a hospital, how seriously ill you are, and the reason ...

Christopher Groux is an editor, writer and accessibility advocate. Before joining the Forbes Health team, he covered Accessibility at Reviewed and gaming at Inverse and Newsweek. When he’s not ...