Several rare disease patient populations received their first-ever FDA-approved drug since Rare Disease Day last year, ...

Northwestern Medicine scientists have uncovered new insights into how neurofilaments act like Velcro in neurodegenerative ...

Enzyme replacement therapy has been successful for some lysosomal storage diseases, for example. But for galactosemia, where ...

For Zevra Therapeutics, last year’s approval of the rare lysosomal storage disorder drug Miplyffa appears to be the gift that ...

Investigators have found that the gene Asah1 plays a crucial protective role in preventing the progression of nonalcoholic fatty liver diseases (NAFLD) into more severe forms of liver disease by ...

Zevra Therapeutics (ZVRA) announced, on Rare Disease Day, the launch of a new disease state awareness campaign, ‘Learn NPC, Read Between the ...