It’s been just six months since Zevra Therapeutics scored an FD | Niemann-Pick disease type C therapy Miplyffa became ...

Researchers identify the Asah1 gene as a promising target for novel therapies to treat nonalcoholic fatty liver disease ...

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 ...

In a murine FTD model, PBFT02 administration improved lysosomal function and reduced neuroinflammation. Passage Bio (Nasdaq: PASG) is a clinical stage genetic medicines company on a mission to improve ...

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 ...

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

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 keeps on giving. | After receiving a rare pediatric disease priority ...

While transient permeabilization of the lysosomal membrane can have acute beneficial effects, supporting inflammation and antigen cross-presentation, sustained or repeated lysosomal perforations have ...

State Key Laboratory of Polymer Science and Technology, Key Laboratory of Polymer Ecomaterials, Changchun Institute of Applied Chemistry, Chinese Academy of Sciences, Changchun 130022, China School of ...