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

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

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

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

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

I envision Chiesi as a proactive leader driving innovation and collaboration within the rare disease community. Our goal is ...

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

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

Denali Therapeutics (NASDAQ:DNLI – Get Free Report) had its target price reduced by Bank of America from $30.00 to $28.00 in ...

lysosomal acidification, oxysterol and bile acid production, and EC immunoactivation, and was associated with disease severity and mortality in patients with PAH. Computational modelling ...