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 ...

AI chatbots can help users navigate common mental health concerns, but they may struggle to recognize rare conditions and ...

The U.S. adopting a Most Favored Nation drug pricing model would hurt medical innovation. | Opinion ...

For families of children born with alternating hemiplegia of childhood, the questions begin almost immediately: Will it get worse? How long do we have? What should we prepare for? The clearest answers ...

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 ...

Lauren Zeller has a one-in-a-million bleeding disorder, but strangers' blood donations give her a chance to play like other ...

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

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 ...

Jeff Allen's son Lucas was diagnosed in 2019 with creatine transporter deficiency, a gene mutation that deprives the brain and muscles of essential creatine. Patients with CTD are usually diagnosed ...

Venture capital, long the dominant engine of drug development, was never really built for rare disease. The returns simply don’t scale to the fund sizes venture capitalists need to justify the effort.