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

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

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

There’s a good chance that you haven’t heard about a rare condition called chromhidrosis. In a nutshell, the person afflicted with the disease produces colored sweat, from yellow to green, even red ...

Patients who have a particular genetic disease whose symptoms include progressively worsening muscle control were able to walk faster following treatment with an experimental Ionis Pharmaceuticals ...

After years stuck in the “doldrums,” the biopharma sector is in a “very good place” heading into the new year, analysts told BioSpace, with both rare and chronic diseases headlining investor and R&D ...

Brain aneurysms occupy a unique and somewhat paradoxical position in cerebrovascular disease. Although unruptured aneurysms ...

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 ...

The FDA has outlined a new review process for drugs and biologics designed to treat ultrarare genetic diseases that would allow a single-arm trial, plus other supportive data, to serve as pivotal ...

Full pipelines and rising revenues lead rare disease biotechs to restyle themselves as more mature, global biopharma ...