Thirty million Americans have a rare disease. Two-thirds of those 30 million are children. There are more than 10,000 rare ...

GeneDx Holdings WGS recently announced a strategic partnership with Komodo Health that is likely to combine its Infinity ...

After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told ...

| Servier is committing to rare neurology for the long term, using focused science and strategic collaborations to bring new ...

The new CDC recommendation takes a much narrower view of the meningococcal disease threat. Because of "the low incidence ...

When our son Peter was diagnosed with an ultra-rare form of muscular dystrophy at the age of 10, the first question we asked was: Is there a treatment? The answer was no. By the time our daughter ...

The Priority Review Voucher (PRV) program is designed to incentivize rare disease drug development.

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...

Dr. Ali Raja says risk-based vaccines have helped keep meningitis rates low; however, public health experts believe if ...

Rare diseases are defined as conditions affecting fewer than 200,000 people in the US or less than 1 in 2,000 in Europe. 1-2 While each disease is individually rare, collectively, they represent a ...

Scientists at Pacific Northwest Research Institute (PNRI) have overturned a long-held belief in genetics: that inheriting two ...