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

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 ...

A study reveals how glutamate drives tau accumulation in Alzheimer’s and related disorders. Researchers found that blocking ...

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 ...