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

Investigators have found that the gene Asah1 plays a crucial protective role in preventing the progression of nonalcoholic ...

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 ...

Investigators have found that the gene Asah1 plays a crucial protective role in preventing the progression of nonalcoholic ...

Rare Disease Day, observed on February 28th, aims to raise awareness about rare diseases and the challenges faced by patients ...

A new study led by Profs. Fan Kelong and Yan Xiyun from the Institute of Biophysics of the Chinese Academy of Sciences ...

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

The Orphan Drugs Market is experiencing significant growth due to increasing investment in rare disease research, regulatory incentives, and advancements in biotechnology. Orphan drugs are specialized ...

With issues in therapy delivery and a barren trial landscape, short-term chances for a commercial galactosemia treatment are ...

Zevra has sold its US Food and Drug Administration (FDA) priority review voucher (PRV) for $150m to an undisclosed party. The ...

In India, a disease is considered rare if it affects fewer than 1 in 2,500 individuals. The lack of a comprehensive registry ...

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