Connecticut prides itself on strong schools, world-class healthcare, and a commitment to caring for its most vulnerable residents. We are a state that values education, innovation, and community. Yet ...

Despite modern high-throughput sequencing, the genetic cause of most rare movement disorders remains unclear. A research team in Bochum and Tübingen has now solved one piece of the puzzle: The ...

See more of our trusted coverage when you search. Prefer Newsweek on Google to see more of our trusted coverage when you search. When Casey McPherson became a father, his purpose became clear. He ...

Ultragenyx’s Rare Bootcamp gives families a roadmap to help drive research and develop new treatments for rare diseases.

The Food and Drug Administration on Tuesday approved a generic medication for a rare brain disorder, while walking back suggestions by President Donald Trump and other administration officials that ...

Scientists have corrected gene mutations in mice causing an ultra-rare disease by editing DNA directly in the brain with a single injection, a feat with profound implications for patients with ...

Millions live with rare diseases, yet most remain without a clear diagnosis. Advances in genomics, AI, and multi-omics are transforming how we identify and understand these conditions. Global ...

Erectile dysfunction, premature ejaculation, and hypogonadism are common clinical diagnoses. Beyond established guidelines, however, there is a group of rare sexual disorders in men that are often ...

WASHINGTON (7News) — Reporter's Notebook: Every year, thousands of infants in America are diagnosed with devastating genetic diseases through newborn screenings. The encouraging news is that many of ...

Edward G. Neilan from NORD discusses critical efforts to speed up therapy development for rare diseases lacking treatments ...