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

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 ...

Kedrion Biopharma is raising awareness for Plasminogen Deficiency Type 1 (PLGD‑1), a rare genetic disorder, through a series of global virtual and in‑person events held in recognition of Plasminogen ...

A new study has uncovered a genetic cause that may explain why some people develop Kaposi sarcoma despite having no apparent ...

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 ...

Vitamin B12 plays a critical role in DNA repair, nerve function, and healthy cell growth. Long-term deficiency may increase ...

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 ...

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 ...

Beta-ketothiolase deficiency is a rare autosomal recessive disorder that disrupts both ketone body utilisation and isoleucine catabolism. Caused by mutations in genes such as ACAT1, which encodes ...

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.