Alpha1-antitrypsin deficiency, an inherited disorder affecting 100,000 people in the U.S., causes a progressive and incurable ...

Rian McCann is almost seven years old. Her parents, Danielle and Frank described her as kind, sassy, smart and above all, resilient. Rian was born with CDKL5 Deficiency Disorder — which prevents her ...

Credit: Getty Images Research findings support the use of vitamin D supplementation to help slow cognitive decline in Alzheimer dementia and reduce Parkinson disease symptom severity. Although decades ...

Findings showed fenfluramine met the primary endpoint and most key secondary endpoints. Topline data were announced from a phase 3 trial that evaluated adjunctive fenfluramine in patients with cyclin ...

If you have factor XI deficiency, your blood doesn’t clot properly because it lacks specific proteins called “clotting factors.” It causes a mild form of hemophilia and may increase bleeding. Factor ...

A new study suggests that certain genetic diseases may be treatable with carefully matched vitamins, including a deadly ...

When you have iron-deficiency anemia, low iron leads to a lack of red blood cells to carry oxygen to various parts of your body. Sometimes a gastrointestinal (GI) disorder causes this type of anemia.

Collaboration leverages AAV manufacturing expertise to advance a preclinical-stage gene therapy program for a rare pediatric neurological disorder.SAN SEBASTIÁN, Spain and LONDON, May 5, 2026 ...

Inflammatory bowel diseases occur when the delicate balance between the gut, the immune system, and the microbiota is ...