Rare diseases are genetic disorders that affect a small proportion of the population. Despite their rarity, they can be a ...

GeneDx, which specializes in diagnostics for rare and ultra-rare genetic disorders, is helping to get kids the diagnostic attention they need as part of Diagnosis is Power. Currently, one in 10 ...

Dr Sudheendra Rao N R from the Organization for Rare Diseases India reveals that approximately 70 million are affected by ...

Worm tracks show how worms move over time and are the basis for recognising the effects of disease mutations as well as candidate treatments that make disease model worms move like wild type ones. The ...

Backed with $82 million and led by two of BioMarin’s former executives, Mendra plans to acquire rare disease assets and use ...

For many rare disease patients, access to expert care remains out of reach. Families often endure years of misdiagnoses and medical visits before finally receiving an accurate diagnosis. This is often ...

Across America, millions of parents of children with rare diseases are in a race against time, hoping that new treatments will be developed fast enough to save their kids. Thanks to advances in ...

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

This article is part of Nature Outlook: Skin, an editorially independent supplement produced with financial support from LEO Pharma. About this content. KaDee Troop is the mother of seven adopted ...