A new research project focusing on rare genetic mutations that are linked to cystic fibrosis just received $600,000 in ...

Genetically targeted technologies are built on Nobel Prize-winning science. They represent a scientific “quantum leap” that ...

Early research shows a one-time gene therapy has potential for addressing both the liver and lung disease that patients with ...

Rare diseases - a medical condition so obscure that even your doctor has to Google. Despite their name, these diseases ...

The researchers say inherited genetic mutations, older parents and improved survival rates for pre-term babies also ...

There are three main types of ovarian cancer: epithelial, germ cell, and stromal. Learn about each type’s defining ...

Here, experts explain genetics in Alzheimer’s and dementia, including if it’s hereditary, how to lower your risk, and ...

Sirolimus, the active ingredient in Felycin-CA1, at a higher dose is used as an immunosuppressant in human patients receiving ...

A multidisciplinary approach, early diagnosis, and comprehensive caregiver education are crucial in effectively managing ...

Scientists have made a potentially “life-changing” discovery that could pave the way for new drugs to treat Parkinson’s ...

Initiation of dosing in our Phase 1b clinical trial represents an important step in the clinical development of GT-02287 and in Gain’s mission to deliver a disease-modifying therapy to people with ...